| 单词 | dystrophy |
| 例句 | There were tiny babies with club feet, children who had paralyzed limbs from polio, adolescents dying from muscular dystrophy. My Life with the Chimpanzees 1988-03-01T00:00:00Z His friend and publicist Glenn Schwartz said in a statement that the cause was muscular dystrophy. Gilbert Gottfried, Comedian With a Distinctive Voice, Dies at 67 2022-04-12T04:00:00Z Evison did more than just wonder — he wrote a comic, tragic and compelling book about a bereaved father who takes the job of caregiver to a teenager with muscular dystrophy. Author Jonathan Evison on loss, survival and life's epic journeys 2012-09-23T14:00:40Z Since then she has been raising awareness of lymphatic edema: “more people have it than MS, muscular dystrophy, ALS and Aids combined – and nobody knows about it.” Kathy Bates: 'I told Clint that after 50 years, I feel like I've hit the big time' 2020-01-17T05:00:00Z Unlike most actresses who have taken on this part, Ms. Ferris, who has muscular dystrophy, is visibly disabled and spends much of the play in a wheelchair. Review: Dismantling ‘The Glass Menagerie’ 2017-03-09T05:00:00Z Another win for Netflix, this indie comedy stars Paul Rudd as an unemployed father, grieving the loss of his son, who becomes caregiver to a teen with muscular dystrophy. These Are the Sundance Movies That Will Be Big in 2016 2016-02-01T05:00:00Z Gottfried died from recurrent ventricular tachycardia due to myotonic dystrophy type II, a disorder that affects the heart, his publicist and longtime friend Glenn Schwartz said in a statement. Gilbert Gottfried, standup comic and actor, dies at 67 2022-04-12T04:00:00Z Bainbridge Island author Evison hit his stride in this hilarious, sad and affecting story of a bereaved young father who takes a job as caregiver to a teenager with muscular dystrophy. 8 great paperbacks to tote this summer 2013-05-10T20:24:08Z As part of that goal, Tyler is raising money to help cure a rare disorder called Duchenne muscular dystrophy. California 12-year-old readies for Mount Everest climb 2016-03-29T04:00:00Z "Not yet, but I am coming close" adding that he would not be satisfied until he had helped "get the cure for muscular dystrophy." Jerry Lewis tight-lipped on telethon role 2011-07-29T20:51:10Z CRAB, which offers individual and group lessons, family sailing events and competitive regattas, can accommodate pretty much any kind of disability, Bollinger said: blindness, deafness, autism, muscular dystrophy, “really anything you can think of.” ‘A way to find the peace I lost’: This Maryland nonprofit is teaching people with disabilities how to sail boats 2019-10-04T04:00:00Z She hopes the telethon can continue to help raise awareness to therapies for muscular dystrophy and other related neuromuscular diseases. Kevin Hart to host famed telethon long hosted by Jerry Lewis 2020-09-09T04:00:00Z It is by far the highest price ever paid for a wristwatch, and all of the proceeds will be donated to research into muscular dystrophy. World's most expensive watch sells for £24.2m in charity auction 2019-11-11T05:00:00Z Lewis said the funds will allow the MDA to continue funding worldwide research to find treatments and cures for muscular dystrophy and related diseases. Jerry Lewis MDA Telethon raises $58.9 million 2010-09-07T00:46:00Z In his experience at a muscular dystrophy camp, video games were one of the most-sought-after activities, particularly with non-ambulatory campers, because games allowed them full, often supercharged mobility. Can gaming satisfy our pandemic thirst for travel and adventure? 2020-09-03T04:00:00Z “Film has sort of died for actors, especially character actors,” said DJ Qualls, who is delivering the strongest work of his career as a young man fighting muscular dystrophy on FXX’s dark comedy “Legit.” Standouts on ‘Grimm,’ ‘Legit,’ ‘Blue Bloods’ and Other Shows 2014-03-21T20:16:42Z A man who's lost everything he ever loved in life begins a new job as a caretaker for a cynical teenager afflicted with advanced Duchenne muscular dystrophy. Paul Rudd and Selena Gomez bring novel to life in 'Fundamentals,' which starts streaming Friday 2016-06-22T04:00:00Z Sickle cell disease fits into that category, as do cystic fibrosis and Duchenne muscular dystrophy. Once Science Fiction, Gene Editing Is Now a Looming Reality 2020-07-22T04:00:00Z The program matches general-education students with those who have intellectual and other disabilities, ranging from autism to cerebral palsy, muscular dystrophy, and visual or hearing impairments. Best Buddies program helps high school students maintain connection during pandemic 2021-04-23T04:00:00Z In this local realm she has created photographs of lush Southern landscapes and studies of her husband’s body revealing the effects of his progressive muscular dystrophy. Sally Mann Turns Her Lens to Friendship and Loss 2016-09-06T04:00:00Z These are single-gene disorders such as muscular dystrophy, cystic fibrosis, sickle-cell disease and Tay-Sachs syndrome but they are the exception. Identically Different: Why You Can Change Your Genes by Tim Spector – review 2012-08-08T09:30:03Z Wong, an outspoken activist diagnosed with a form of muscular dystrophy when she was 2, created the hashtag #CrippingTheMighty to respond to the site’s questions while drawing a larger audience’s attention. A disability-focused website ran a ‘funny’ post on autism. Anger ensued. 2016-01-05T05:00:00Z Growing up with muscular dystrophy, she rarely saw physically disabled actors on TV at all. Jillian Mercado on ‘Generation Q’ and the Importance of Joyful Stories 2021-10-04T04:00:00Z And, to name just a few hidden disabilities: multiple sclerosis, muscular dystrophy, asthma, arthritis, tendinitis, heart conditions, recent illness or surgery . Carolyn Hax: When elevator users push someone’s buttons the wrong way 2016-07-15T04:00:00Z SAT Walk any distance to raise funds for Muscular Dystrophy Association for children and adults living with muscular dystrophy, ALS and related diseases, 9:30 a.m. Community calendar 2012-02-29T23:02:08Z In 1976, moody Robert Carter goes somewhat neglected by his mother and father, who run the local theme park, as they care for his brother, Liam, who has muscular dystrophy. Review | ‘Setting Free the Kites,’ by Alex George 2017-02-26T05:00:00Z How did you incorporate your experiences as a queer, Hispanic person with muscular dystrophy into Maribel’s character while also being sensitive to your own boundaries and privacy? Jillian Mercado on ‘Generation Q’ and the Importance of Joyful Stories 2021-10-04T04:00:00Z A sad, funny and highly entertaining novel about a man engulfed by loss who becomes caregiver to a teenager with muscular dystrophy. Alexie, Evison, Wilson are Pacific Northwest Book Award winners 2013-01-07T21:40:18Z Ms. Spencer, who has muscular dystrophy and uses a wheelchair, also creates videos debunking disability myths and shares fashion and dating tips on Instagram and YouTube. Lauren Spencer Is a Sex-Positive Disability Influencer 2022-12-23T05:00:00Z Though he gets about in a motorized scooter, he has not let his myotonic dystrophy, a disease involving progressive muscle loss, curtail his travels. For the Disabled Traveler, Strategies for a Successful Trip 2017-02-03T05:00:00Z “He has very high levels of creatine kinase … this points to muscular dystrophy … and by virtue of his age and sex, it’s statistically most likely he has Duchenne.” Our beautiful sons could die before us 2014-08-16T04:00:00Z Meagu's father was badly affected by a condition called Charcot-Marie-Tooth disease, a form of muscular dystrophy, and died in his 50s. IVF technique that tests embryos for genetic disorders has first success 2014-07-27T04:00:00Z At length she returns to her children, ever more liable to time’s assaults, to her own close brush with death in a riding accident, and to the sad progress of her husband’s late-onset muscular dystrophy. Sally Mann’s Haunted South 2018-03-29T04:00:00Z A 17-year-old Israeli girl who has myotonic dystrophy, Rut cannot walk and uses a wheelchair. For the Most Vulnerable Hostages, a Plea for Mercy 2023-10-21T04:00:00Z FDA leaders have recently emphasized a new level of “regulatory flexibility” when reviewing experimental treatments for fatal, hard-to-treat conditions, including ALS, Alzheimer’s and muscular dystrophy. FDA advisers vote against experimental ALS treatment pushed by patients 2023-09-27T04:00:00Z Alvin was diagnosed with muscular dystrophy and cared for on the neonatal unit following his birth. Lucy Letby: Deeside mum haunted by photo with baby before death 2023-08-31T04:00:00Z Another time, according to the filing, Lewis gleaned some closed-door information about a muscular dystrophy drug company in which he was a major investor. British billionaire, owner of Tottenham soccer team, arrested on insider trading charges 2023-07-26T04:00:00Z For example, a different gene could be used to treat Fuchs’ dystrophy, which affects 18 million people in the U.S. and accounts for about half the nation’s corneal transplants. Gene therapy eyedrops restored a boy’s sight. Similar treatments could help millions 2023-07-24T04:00:00Z Parents of children living with muscular dystrophy say better expertise is needed in Wales to treat it. Muscular dystrophy: Child racks up 1,200 miles a month for treatment 2023-07-04T04:00:00Z The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids 2023-06-22T04:00:00Z After that, she will go back to the day job of running Duchenne UK, the charity she set up after Eli was diagnosed with muscular dystrophy 13 years ago. 'My muscle condition won't stop me playing Glastonbury' 2023-06-21T04:00:00Z But inspired by an older man who was missing nearly half of the protein yet had only mild muscular dystrophy, Chamberlain’s lab 2 decades ago devised a gene for a miniature dystrophin. Muscular dystrophy gene therapy nears approval, but safety concerns linger 2023-05-23T04:00:00Z There were five people in the building with disabilities including muscular dystrophy, say support groups. Israel-Gaza: Palestinian family left homeless after strikes 2023-05-17T04:00:00Z The genetic defects can result in diseases such as muscular dystrophy, epilepsy, heart problems and intellectual disabilities. Britain: 1st babies born in country using DNA from 3 people 2023-05-10T04:00:00Z Sessions grew especially attached to the dogs, which was tough: Some were bred for two different forms of muscular dystrophy, one 100% fatal. Suffering in silence: Caring for research animals can take a severe mental toll 2023-03-09T05:00:00Z Mr. Thorleifsson has muscular dystrophy, which makes it difficult to perform physical tasks. Elon Musk apologizes for mocking fired disabled Twitter employee 2023-03-08T05:00:00Z Mr Thorleifsson has muscular dystrophy and has campaigned in Iceland for better wheelchair access. Elon Musk apologises to Twitter worker over online row 2023-03-07T05:00:00Z For example, He, after leaving prison last year, set up a nonprofit institute to work on Duchenne muscular dystrophy and other topics. In wake of gene-edited baby scandal, China sets new ethics rules for human studies 2023-03-06T05:00:00Z Mr. Thorleifsson, who sold his digital brand agency to Twitter in 2021, has muscular dystrophy, which severely limits a person’s ability to perform daily physical activities. Musk publicly mocks Twitter employee for asking if the CEO fired him 2023-03-07T05:00:00Z “They make it all seem less arbitrary,” says Sessions, whose work with the muscular dystrophy dogs often left him more upset than enlightened. Suffering in silence: Caring for research animals can take a severe mental toll 2023-03-09T05:00:00Z "I decided to sell for a few reasons but one of them is that I have muscular dystrophy and my body is slowly but surely failing me," he told the BBC. Have I been fired? worker asks chief executive Elon Musk on Twitter 2023-03-07T05:00:00Z A new piece of technology has been created for the benefit of thousands of children in the UK living with muscular dystrophy. 'I hope this new suit will help me live independently' 2023-02-28T05:00:00Z She left with her fiancé Jared Clark and his son, who is afflicted with myotonic dystrophy and is autistic. Drink bottled water, officials tell Ohio town hit by toxic train crash 2023-02-14T05:00:00Z “Individuals with special needs can be on a board. They can contribute. They can give you a perspective,” said Zaman, 37, who has muscular dystrophy. ‘Not just the ramp.’ Worship spaces need more accessibility 2022-12-29T05:00:00Z He claims that he could develop a cure for the degenerative genetic disease Duchenne muscular dystrophy – if he receives funding in excess of US$100 million. Documentary spurs a new look at the case of the first gene-edited babies 2022-12-27T05:00:00Z The app learns how to better recognise speech patterns of those who might struggle - like people with muscular dystrophy - and helps them communicate more easily. Google opens its first UK centre making tech for disabled people 2022-12-03T05:00:00Z The progressive life-limiting condition causes all the muscles in the body to gradually weaken, but a new suit allows those living with muscular dystrophy to maintain the use of their arms. 'I hope this new suit will help me live independently' 2023-02-28T05:00:00Z He had cystic fibrosis and Duchenne muscular dystrophy, among other conditions. Benefit cuts for disabled people in hospital called unfair 2022-11-02T04:00:00Z She had a learning disability and lived with a serious muscle condition called myotonic dystrophy. Tommy Jessop: Why I investigated care for patients like me 2022-10-09T04:00:00Z But that is impossible for Timothy Carey, an Appleton, Wisconsin, resident who has Duchenne muscular dystrophy and lives on a ventilator. Court victories deliver cautious hope for voters with disabilities 2022-09-16T04:00:00Z Alice Wong was born with muscular dystrophy, which hampered her ability to walk by the time she was 7 or 8 years old. Review | How disability advocate Alice Wong turned her anger into action 2022-09-09T04:00:00Z Timothy Carey, 49, who has Duchenne muscular dystrophy and lives in Appleton, Wisconsin, is one of four plaintiffs listed in the lawsuit. Forbidden from getting help returning absentee ballots, disabled voters sue Wisconsin 2022-07-26T04:00:00Z Among those bringing the lawsuit is Timothy Carey, who is unable to move his body because he has Duchenne muscular dystrophy. After Wis. Supreme Court absentee ballot decision, disabled people sue 2022-07-25T04:00:00Z One of them, Timothy Carey, has Duchenne muscular dystrophy, and is unable to move his body and is on a ventilator. Wisconsin disabled voters file federal lawsuit over ballots 2022-07-25T04:00:00Z Diseases transmitted by X-linked recessive inheritance include color blindness, the blood-clotting disorder hemophilia, and some forms of muscular dystrophy. Anatomy and Physiology 2013-06-19T00:00:00Z Morgan Hoffmann, who is fighting facioscapulohumeral muscular dystrophy, finished at 2 over in his third and final tournament of the year on a three-event medical exemption. Schauffele wins at Travelers after Theegala’s double bogey 2022-06-26T04:00:00Z The 68-year-old, who is president and chief executive of the disability rights group Not Dead Yet, has a form of muscular dystrophy and uses her ventilator roughly 22 hours each day. These machines to help people breathe were recalled a year ago. Many still use them 2022-06-23T04:00:00Z Morgan Hoffmann, making a long-shot bid to keep his tour card after two years away from golf because of muscular dystrophy, missed the cut with rounds of 73 and 80. Jason Day handles rain, expands lead at Wells Fargo 2022-05-06T04:00:00Z NOTES: Morgan Hoffmann, who put his career on hold after he was diagnosed with muscular dystrophy, is making one of two remaining starts on a major medical extension. ‘Good vibes’ for McIlroy as he defends at soggy Wells Fargo 2022-05-04T04:00:00Z The eldest of three children, he was born with muscular dystrophy, a disease that causes weakness and loss of muscle mass. War in Ukraine: A family's struggle to get to Scotland 2022-04-24T04:00:00Z The performer — revered for his irreverent and shocking style of comedy — also had myotonic dystrophy, a muscular disease characterized by an inability to relax muscles after they contract. Comedian Gilbert Gottfried was diagnosed with a heart condition before his death 2022-04-13T04:00:00Z His friend and publicist Glenn Schwartz said in a statement he died from recurrent ventricular tachycardia due to myotonic dystrophy type II, a disorder that affects the heart. Gilbert Gottfried: Stars pay tribute to late comedian and actor 2022-04-13T04:00:00Z The cause was complications from a form of muscular dystrophy that can lead to an irregular heartbeat, said his friend and publicist Glenn Schwartz. Gilbert Gottfried, comedian with voice for controversy, dies at 67 2022-04-12T04:00:00Z The cognitive disorder affects about 2 million Americans, making it more common than Parkinson’s disease, cerebral palsy or muscular dystrophy, according to the National Aphasia Assn. What is aphasia? Behind the common cognitive disorder that has affected Bruce Willis 2022-03-30T04:00:00Z A girl with muscular dystrophy who was on a breathing machine and her mother were inside their one-story house when it was picked up off its foundation and tossed about 30 feet. South Carolina residents trapped, Louisiana declares state of emergency after tornadoes 2022-03-24T04:00:00Z One day after his 30th birthday, Vincent was diagnosed with muscular dystrophy. Illinois man fulfilling sports viewing dreams despite MD 2022-02-07T05:00:00Z She has facioscapulohumeral muscular dystrophy, a disorder mostly affecting the muscles of the face, shoulder blades and upper arms. Covid: Animal hydrotherapy access 'better than people's' 2021-12-23T05:00:00Z He recalls attending a muscular dystrophy meeting where he said he intended to defy his genetics and use IVF to have children without the disease. IVF used by some to avoid passing on genetic diseases to offspring 2021-12-03T05:00:00Z My brother Scott was born with muscular dystrophy. Long-term care: Ensure respect and dignity 2021-12-03T05:00:00Z “I’m used to the look on people’s faces when they can’t understand what I’ve said,” Aubrie Lee, a brand manager at Google whose speech is affected by muscular dystrophy, said in a blog post today. Google is taking sign-ups for Relate, a voice assistant that recognizes impaired speech 2021-11-09T05:00:00Z Although he managed to correct muscular dystrophy in mice — a feat reported at the same time by two other labs — that was no guarantee the gene therapy would work in humans. He can’t cure his dad. But a scientist’s research may help everyone else 2021-11-07T04:00:00Z Karine Elharrar - who has muscular dystrophy - said she was forced to return to her hotel 50 miles away on Monday after waiting outside for two hours. COP26: Has the Glasgow climate summit been poorly organised? 2021-11-04T04:00:00Z The UK's Environment Secretary, George Eustice, said the incident was "deeply regrettable" and the government had apologised to Ms Elharrar - who has muscular dystrophy. COP26: UK apologises to Israeli minister for wheelchair access problems 2021-11-02T04:00:00Z Michael Johnson was born with a form of muscular dystrophy that caused respiratory problems and required him to use a ventilator. NBA broadcaster Ernie Johnson's son, Michael, dead at 33 2021-10-31T04:00:00Z “We are, as a species, wildly optimistic about ourselves,” says Peay, who works with patients and families with the progressive muscle disorder Duchenne muscular dystrophy. We Need to Ground Truth Assumptions about Gene Therapy 2021-10-22T04:00:00Z More common muscular dystrophies are caused by a mutation that leaves patients lacking a particular protein. He can’t cure his dad. But a scientist’s research may help everyone else 2021-11-07T04:00:00Z Still, even limited advances pave the way for ongoing progress, she adds, pointing to research in her patients who have Duchenne muscular dystrophy: “In most of these clinical trials, we learn important things.” Four Success Stories in Gene Therapy 2021-10-20T04:00:00Z Fortunately, the degenerative nerve disease is "not deadly," he says, but likened it to the toll muscular dystrophy and Parkinson's disease has on patients. Country star Alan Jackson opens up about health woes in rare interview: 'I'm stumbling around stage now' 2021-09-28T04:00:00Z One parent told the U.K. consultation that he was glad not to know his son had Duchenne muscular dystrophy before symptoms appeared—they had four happy, worry-free years. Sequence every newborn’s DNA? Despite obstacles, UK pushes ahead 2021-09-22T04:00:00Z A handful of gene-targeted treatments are under development for sickle cell disease, and hundreds more are being investigated for a variety of conditions, including cystic fibrosis, muscular dystrophy, hemophilia, Huntington’s disease, HIV and cancer. We Need to Ground Truth Assumptions about Gene Therapy 2021-10-22T04:00:00Z After he graduated from Harvard, Tabebordbar thought he had a chance to develop a gene therapy for muscular dystrophy at a biotech company. He can’t cure his dad. But a scientist’s research may help everyone else 2021-11-07T04:00:00Z Mutations in RPE65 are just one cause of inherited retinal dystrophy, but it was a cause that Bennett and Maguire could act on. Four Success Stories in Gene Therapy 2021-10-20T04:00:00Z And after one severe episode during which he was hospitalized for weeks, doctors worried he had muscular dystrophy. This genetic sleuth has uncovered a new category of disease marked by sporadic fevers and inflammation 2021-09-22T04:00:00Z Mr King, who has been going to Leeds games since he was a child, was diagnosed with macular dystrophy at age 12. Leeds Rhinos: Blind rugby league player makes conversion 2021-09-21T04:00:00Z The children had various conditions affecting the brain or nervous system, including cerebral palsy and muscular dystrophy. Covid jab side-effects 'mild' for at-risk children 2021-08-26T04:00:00Z That includes muscular dystrophies that affect about 100,000 people in the United States, according to the Muscular Dystrophy Association. He can’t cure his dad. But a scientist’s research may help everyone else 2021-11-07T04:00:00Z She was irritated at being dragged to muscular dystrophy events where adults would tell her how brave she was. How a teen punk led a movement for disabled people online 2021-07-27T04:00:00Z Due to the pandemic he currently teaches online, mostly children with cerebral palsy, but the program is also designed for those with muscular dystrophy, limb amputation, quadriplegia or spinal cord injury. Technology helps disabled student play the harp with her eyes 2021-06-17T04:00:00Z It’s also being studied to treat cancer, muscular dystrophy and other diseases. Answers to Your Questions About Covid Vaccines and Kids 2021-05-21T04:00:00Z In 2016, the FDA approved a drug for Duchenne muscular dystrophy after outside experts recommended against approval and internal reviewers raised doubts about the drug’s efficacy. Alzheimer’s drug sparks emotional battle as FDA nears deadline on whether to approve 2021-05-31T04:00:00Z More detailed and sophisticated testing finally revealed the answer: His father has an extraordinarily rare genetic disorder, facioscapulohumeral muscular dystrophy, or FSHD, that affects an estimated four to 10 out of every 100,000 people. He can’t cure his dad. But a scientist’s research may help everyone else 2021-11-07T04:00:00Z The drug developer reported encouraging results from a study on a potential muscular dystrophy treatment. Walmart, Sarepta rise; Chevron, D.R. Horton fall 2021-05-18T04:00:00Z The sisters both have weakened lungs from muscular dystrophy and have stayed home for more than a year, tended to by Socorro’s husband, Martin Martinez. Nurses, nonprofits, others take vaccine to homebound people 2021-05-12T04:00:00Z The real promise of Crispr is for treating diseases caused by genetic mutations, from muscular dystrophy to congenital blindness, and even some cancers. After the Nobel, what next for Crispr gene-editing therapies? 2021-02-21T05:00:00Z The drug developer reported mixed study results for a potential muscular dystrophy treatment. Sarepta, Newmont fall; WD-40, New Relic rise 2021-01-08T05:00:00Z But after about a year, the company called everyone into a conference room to tell them there was going to be a reorganization and the muscular dystrophy program was being dropped. He can’t cure his dad. But a scientist’s research may help everyone else 2021-11-07T04:00:00Z Nora Voytko, who lives near Austin, Texas, helps care for her adult son-in-law, who is disabled due to muscular dystrophy, a genetic disease that causes progressive weakness and loss of muscle. AP-NORC poll: New angst for caregivers in time of COVID-19 2020-10-13T04:00:00Z Initial attempts should be for serious diseases caused by a single gene, such as muscular dystrophy, cystic fibrosis, the blood disorder beta thalassemia and Tay-Sachs, a neurological disease, the report says. Still too soon to try altering human embryo DNA, panel says 2020-09-03T04:00:00Z Duchenne muscular dystrophy is a severe neuromuscular disorder that limits life expectancy. Photos document 'hidden' life with a disability 2020-08-25T04:00:00Z Bell has a rare form of muscular dystrophy that affects her strength and balance, making it harder for her to do things like walk on her own. Lawsuit: School mishandled disabled student’s rape report 2020-08-05T04:00:00Z The cutbacks hit close to home for Furlong, whose two sons died from muscular dystrophy in their teens. COVID-19 cancels charity galas and walks. Science is paying the price 2020-06-24T04:00:00Z Delashmit, who was indicted in March, is accused of faking illnesses to attend Camp Summit in Dallas, which serves people with muscular dystrophy and spinal muscular atrophy, between October 2015 and March 2016. Woman claiming breast cancer, other issues accused of fraud 2020-06-22T04:00:00Z The sisters suffer from myotonic dystrophy, a debilitating, degenerative genetic disorder affecting muscle function and mental processing. Judge sides with Guam resident in Social Security case 2020-06-22T04:00:00Z Ramy, the character, has an anti-Semitic uncle, a mother whose ignorance results in unintentional bigotry, and a best friend with muscular dystrophy who is often incredibly selfish. Hulu’s Ramy takes cringe comedy to a dark and funny place 2020-05-29T04:00:00Z The same month she was yanked from the car, she came to the United States to attend a conference for people with muscular dystrophy. Perspective | In Cameroon, she was a journalist and disability activist. In the U.S., she’s stuck in place. 2020-05-27T04:00:00Z Blumenthal put up a poster of a boy named Connor who he said was one of his constituents, who suffers from muscular dystrophy. Senate Dems attack Trump judicial nominee over past comments on ObamaCare in tense hearing 2020-05-06T04:00:00Z Even more remarkable is that the young Rollman has muscular dystrophy and does his shooting from a wheelchair. Pandemic postpones national Paralympics trials for ND teen 2020-04-13T04:00:00Z Some FDA observers defend the agency by pointing out it has occasionally given full commercial approval to drugs based on relatively scant evidence, including a controversial treatment for Duchenne muscular dystrophy last year. Former FDA leaders decry emergency authorization of malaria drugs for coronavirus 2020-04-07T04:00:00Z Many were priced well above $100,000 for a year’s supply, including drugs for cancer, muscular dystrophy and other genetic disorders. Potential coronavirus drug granted ‘rare disease treatment’ status 2020-03-24T04:00:00Z She has muscular dystrophy and relies on a wheelchair to get around. Perspective | In Cameroon, she was a journalist and disability activist. In the U.S., she’s stuck in place. 2020-05-27T04:00:00Z Hoffman was diagnosed in 2016 with a form of muscular dystrophy. Cristie Kerr brings her views to TV at Honda Classic 2020-02-25T05:00:00Z It revealed that Hoffmann was suffering from the progressing effects of facioscapulohumeral muscular dystrophy. There's no stopping Morgan Hoffmann in his fight against muscular dystrophy - Golf Digest 2020-02-24T05:00:00Z In a paper published last year6, Mali and his colleagues injected guide RNAs into mice born with a genetic mutation that causes muscular dystrophy. Step aside CRISPR, RNA editing is taking off 2020-02-03T05:00:00Z Thankfully, Bream eventually found a doctor who was able to diagnose her condition as chronic erosion syndrome exacerbated by corneal map-dot-fingerprint dystrophy. Shannon Bream shares 'darkest days' to 'hopefully bring someone else back from the brink' 2020-01-23T05:00:00Z At one point, the musician was thought to have developed muscular dystrophy as a result of the interrogations he endured. Mehdi Rajabian: Risking his life to make music in Iran 2020-01-22T05:00:00Z The most recent involved an experimental treatment called Stamina therapy, developed by an entrepreneur who claimed to be able to regenerate nerves from stem cells and cure conditions such as Parkinson's disease and muscular dystrophy. This Italian scientist has become a celebrity by fighting vaccine skeptics 2020-01-02T05:00:00Z Facioscapulohumeral muscular dystrophy causes atrophy of the chest, back, neck, arms and sometimes legs. There's no stopping Morgan Hoffmann in his fight against muscular dystrophy - Golf Digest 2020-02-24T05:00:00Z A handful of start-up companies are beginning to use RNA-editing systems to develop potential treatments for everything from genetic diseases such as muscular dystrophy to temporary maladies such as acute pain. Step aside CRISPR, RNA editing is taking off 2020-02-03T05:00:00Z A funny and heartbreaking story of a man wrestling with an unendurable family tragedy who becomes a caretaker to a teenager with Duchenne muscular dystrophy. The 2010s in books: 10 titles from Washington-state authors that defined the decade 2019-12-23T05:00:00Z I have a rare form of muscular dystrophy, and can’t walk long distances. Ways to make meetings accessible 2019-12-17T05:00:00Z After working as a bouncer and as an aide to a man with muscular dystrophy, Mr. Semexan knew he wanted to find work in education. He Helps Parents Get a Second Chance at Learning 2019-12-16T05:00:00Z The drug developer received regulatory approval for a muscular dystrophy treatment. Oracle and Tapestry slip while Adobe and Sarepta advance 2019-12-13T05:00:00Z In 2016, FDA leaders cleared the company’s first muscular dystrophy drug, overruling agency reviewers who said there was little evidence it worked. In surprise decision, US approves muscular dystrophy drug 2019-12-13T05:00:00Z His unproven technique claimed to convert bone-marrow cells into neuronal stem cells for the treatment of neurological conditions such as Parkinson’s disease and muscular dystrophy. The conceptual mistake that lies at the heart of economics 2019-12-12T05:00:00Z Both born severely disabled by cerebral palsy and muscular dystrophy, the severity of the boys’ condition is on stark display. 'Bhopal’s tragedy has not stopped': the urban disaster still claiming lives 35 years on 2019-12-08T05:00:00Z The therapy is designed to treat a form of Duchenne muscular dystrophy caused by mutations in the RNA that codes for the protein that helps to connect muscle fibers’ cytoskeletons to a surrounding matrix. Gene Therapy Arrives 2019-12-03T05:00:00Z There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. Pfizer, Novartis lead pharma spending spree on gene therapy production 2019-11-27T05:00:00Z Auction house Christie's, which ran the sale for charity to benefit muscular dystrophy, said the Grandmaster Chime reference 6300A-010 was created solely for the event. Rare Patek Phillipe watch sells for $31M at auction 2019-11-11T05:00:00Z Heart failure is a particular problem for people with muscular dystrophy, Dr. Lee noted. This Treatment Can Cure Cancer. Can It Mend the Heart? 2019-09-11T04:00:00Z Inherited retinal dystrophies cause the gradual degeneration of the light-sensitive cells on the retina. NHS to offer gene therapy for rare eye disease 2019-09-03T04:00:00Z The Food and Drug Administration declined approval for the drug developer’s newest muscular dystrophy treatment. Home Depot and Moderna rise while Fabrinet and Sarepta fall 2019-08-20T04:00:00Z Over the past two years, Mark Honey’s rare form of muscular dystrophy has proved so debilitating that he has lost control of his hands, legs and arms. ‘This will be catastrophic’: Maine families face elder boom, worker shortage in preview of nation’s future 2019-08-14T04:00:00Z Mindy Scheier, on the other hand, was a career fashion designer facing an urgent plea for jeans from her son Oliver, then 8, whose muscular dystrophy required leg braces. For people with disabilities, finding clothing that is stylish and meets their needs is getting easier. 2019-08-02T04:00:00Z The connection also fashioned a new friendship between her designer, Kylie McRobie of Scarsdale, New York, and Victoria, who has muscular dystrophy and requires a wheelchair for mobility. Girl rocks custom ‘adaptive’ prom dress made by fellow teen 2019-06-14T04:00:00Z Gene therapies are also being tested to treat hemophilia and Duchenne muscular dystrophy, another rare disorder. For Anderson family, an early bet on SMA gene therapy 2019-05-24T04:00:00Z The FDA is poised to review more applications for gene therapies for inherited diseases such as hemophilia and Duchenne muscular dystrophy. The costliest drug on the planet will treat infants with rare disease. The market fight focused on cost and safety is just getting started. 2019-05-15T04:00:00Z And 60 days after a similar treatment, patients with a disease called limb-girdle muscular dystrophy are making substantial amounts of a missing muscle protein, Sarepta recently reported. Boys with a rare muscle disease are breathing on their own, thanks to gene therapy 2019-05-02T04:00:00Z New drugs that may fall under their scrutiny in the next year include potentially life-saving therapies for spinal muscular atrophy and Duchenne muscular dystrophy as well as oral treatments for migraine, diabetes and multiple sclerosis. The obscure advisory committees at the heart of the U.S. drug... 2019-04-29T04:00:00Z Scheier, a Livingston resident, founded Runway of Dreams in 2014 after trying and failing to find adaptive fashions for her son, Oliver, who also has muscular dystrophy. Girl rocks custom ‘adaptive’ prom dress made by fellow teen 2019-06-14T04:00:00Z Dr. Phil A woman is accused of faking terminal cancer, pregnancies, miscarriages and muscular dystrophy. What's on TV Friday: 'Blindspotting' on Cinemax 2019-04-25T04:00:00Z The drug developer reported that some patients in an early study of a potential muscular dystrophy drug experienced some severe side effects. Qualcomm and Progressive jump while Wave Life Sciences drops 2019-04-16T04:00:00Z Gold cast Madison Ferris, a severely disabled actress, who has muscular dystrophy, in the role. Sam Gold’s Self-Serving Vision of “King Lear” 2019-04-08T04:00:00Z The lawsuit was filed by the father of a fan with a form of muscular dystrophy. Wrigley Field renovation adds wheelchair-accessible seating 2019-04-03T04:00:00Z Grit alone got Linda Greene through her husband’s muscular dystrophy, her daughter’s traumatic brain injury, and her own mysterious illness that lasted for three years and left her vomiting daily before doctors identified the cause. Can genetic testing help doctors better prescribe antidepressants? There’s quite a debate. 2019-03-29T04:00:00Z I was surprised and moved, for example, to learn from a BBC report about a young man named Mats Steen whose body was severely disabled by Duchenne muscular dystrophy. Opinion | This Friendship Has Been Digitized 2019-03-23T04:00:00Z He later died of complications from muscular dystrophy. Boise State faces challenge in Oregon State to open NCAAs 2019-03-22T04:00:00Z He had complications from a fall and from an adult-onset form of muscular dystrophy. William C. Powers Jr., author of report outlining Enron corruption, dies at 72 2019-03-15T04:00:00Z The cause was complications of a fall and of oculopharyngeal muscular dystrophy, a rare, adult-onset muscle disorder. This week’s passages 2019-03-15T04:00:00Z Most of the scientific community strongly supports editing somatic cells to treat diseases including sickle cell anemia, Huntington’s disease and Duchenne muscular dystrophy. Scientists Call for a Moratorium on Editing Inherited Genes 2019-03-13T04:00:00Z The university said Powers died Sunday in Austin from complications from a fall several months earlier and oculopharyngeal muscular dystrophy, a rare adult-onset muscle disorder. Former UT President Bill Powers dies at 72 2019-03-10T05:00:00Z People donate money for muscular dystrophy or Alzheimer’s or Parkinson’s research, not so much for the vague notion of disability rights. Opinion | Disability and disease aren’t interchangeable 2019-03-07T05:00:00Z A Realtor now, Carson still sculpts waterfowl, mostly for fundraisers like those that combat Duchenne muscular dystrophy, a progressive disease that afflicts his son, Mark, 17. For this waterfowl artisan, carving duck decoys is old hat 2019-02-25T05:00:00Z But as the hand-held controllers became more complex and her muscular dystrophy became more debilitating, she was eventually forced to stop playing console games. Adaptive Video Game Controllers Open Worlds for Gamers With Disabilities 2019-02-20T05:00:00Z Labour’s Lyn Brown asks about a drug used to treat muscular dystrophy. PMQs: May faces Corbyn after ERG refuse to back government Brexit motion - Politics live 2019-02-13T05:00:00Z In 1998, when she was 13, Brandy was found to be suffering from reflex sympathetic dystrophy syndrome, a rare nerve affliction with no known cure. ‘We all have bills. We all have to eat.’ For furloughed federal workers, the first missed paychecks ratchet up anxiety. 2019-01-11T05:00:00Z First, Morgan Hoffmann, whose career has unfortunately been put on hold due to muscular dystrophy, said his engagement “took 2018 from being the worst, to the absolute best year of my life!” Golf’s controversial new rule, a pair of tour pro marriage proposals, and a petition you need to sign now - Golf Digest 2019-01-08T05:00:00Z You don’t need to know the melancholy of Larry’s late-onset muscular dystrophy, recorded in other portrait photographs here, to read this as a walk through a valley of the shadow of death. Review: A child in 'The Ditch' and other mesmerizing moments in 'Sally Mann: A Thousand Crossings' at the Getty 2019-01-02T05:00:00Z The former World No. 1 amateur golfer whose playing career was interrupted after being diagnosed with muscular dystrophy late in 2016 got engaged to girlfriend Chelsea Colvard on a boat in the Bahamas. Morgan Hoffmann gets engaged, transforming 2018 from "the worst, to the absolute best year of my life" - Golf Digest 2019-01-02T05:00:00Z Ostarine is currently being researched as a treatment for a variety of muscle-wasting diseases such as osteoporosis, muscular dystrophy and chemotherapy in cancer patients. Three Clemson players fail drug tests ahead of Cotton Bowl 2018-12-24T05:00:00Z Unlike disorders caused by mutations in a single gene — such as cystic fibrosis or some types of muscular dystrophy — neuropsychiatric disorders including schizophrenia involve hundreds of genes that interact with environmental factors. Huge brain study uncovers ‘buried’ genetic networks linked to mental illness 2018-12-12T05:00:00Z One experiment conducted by the Japan Aerospace Exploration Agency looked at the proteins associated with Duchenne muscular dystrophy, which primarily affects young boys and leaves them immobile. Startups defy gravity to explore space's commercial frontiers 2018-11-27T05:00:00Z Scientists are investigating the use of CRISPR-Cas9 as a treatment for individuals suffering from genetic diseases, such as muscular dystrophy and sickle cell disease. CRISPR bombshell: Chinese researcher claims to have created gene-edited twins 2018-11-26T05:00:00Z Occurring mostly in males, it is the most common type of muscular dystrophy, striking about one in 3,500 boys and causing their muscles to start breaking down in early childhood. CRISPR Gene Editing Shows Promise for Treating a Fatal Muscle Disease 2018-10-29T04:00:00Z For several years he had suffered from a rare form of muscular dystrophy but he continued to work. Rachid Taha obituary 2018-09-13T04:00:00Z The exhibit also includes portraits of people with genetic conditions such as a form of muscular dystrophy and black-and-white photographs of scars. What it feels like to step inside someone’s skin 2018-08-31T04:00:00Z She played rugby and hockey in her youngest years but, by 11, she developed a pain in her knees that was later attributed to reflex sympathetic dystrophy. Hilary Lister, quadriplegic yachtswoman who sailed solo across English Channel, dies at 46 2018-08-29T04:00:00Z He was surrounded by patients with incurable diseases, including a second grader with Duchenne muscular dystrophy, who got up from his small wheelchair to hug the president. The So-Called Right to Try Law Gives Patients False Hope 2018-09-01T04:00:00Z Tway began his week playing in a charity event with good friend Morgan Hoffmann, who is coping with muscular dystrophy. 4-way tie for lead at Northern Trust as Woods stalls 2018-08-23T04:00:00Z A progressive neurological disorder, reflex sympathetic dystrophy, left Ms Lister in a wheelchair from the age of 15. Record-breaking quadriplegic sailor dies 2018-08-20T04:00:00Z Swimming with sharks at England’s Blue Planet Aquarium is a popular charity event in the United Kingdom that has raised money for disabled children and people with muscular dystrophy. I’m Running in the Serengeti With Lions—Please Donate 2018-08-13T04:00:00Z Third, for the disease reports, my eye lit on the phrase “variants not detected” for Parkinson's disease, cystic fibrosis, muscular dystrophy, sickle cell anemia, Tay-Sachs and, most concernedly, Alzheimer's. What Personal Genome Testing Can and Can't Do 2018-07-31T04:00:00Z Further ahead, there is hope that CRISPR-Cas9 will help treat diseases such as AIDS, cystic fibrosis, Huntington’s chorea and Duchenne muscular dystrophy. The safety of CRISPR-Cas9 gene editing is being debated 2018-07-19T04:00:00Z She was also diagnosed with a rare form of muscular dystrophy - a muscle wasting disease with no cure - aged four. Asos praised for disabled-friendly clothes 2018-07-05T04:00:00Z Some welcome news arrived recently for boys with a condition called Duchenne muscular dystrophy. A Risky Drug Approval Lesson 2018-07-04T04:00:00Z In 2016, internal reviewers and an advisory committee called for rejecting a drug for a rare muscular disease called Duchenne muscular dystrophy. FDA repays industry by rushing risky drugs to market 2018-07-02T04:00:00Z Sarepta Therapeutics Inc. gained about $2.5 billion of stock market value Tuesday after an early stage trial of a gene therapy showed promise in its ability to treat patients with Duchenne muscular dystrophy. Sarepta Gets Boost From Early Trial Results on Muscular Dystrophy Treatment 2018-06-19T04:00:00Z Since Rob was a child, Bob added, he had helped to care for his sister, who had muscular dystrophy and cerebral palsy. Letter laid groundwork for Lennox murder-suicide 2018-05-12T04:00:00Z But the website lists dozens of diseases, including congestive heart failure, muscular dystrophy and stroke that Cell Surgical Network is “currently studying.” Stem-cell clinics spread fast but therapies mostly unproved 2018-04-29T04:00:00Z After dealing with the muscle spasms, fatigue and increasing weakness from muscular dystrophy that was first diagnosed when she was 16, she was ready to try something different. Budtenders to be in high demand in Michigan 2018-04-08T04:00:00Z Gene therapies for several other inherited diseases are already in the pipeline, including against sickle cell disease and a type of muscular dystrophy. Medical breakthrough: Seattle man is first adult to get new gene therapy for vision 2018-04-01T04:00:00Z As his findings were nearing publication earlier this month, Wilson resigned from the board of Solid Biosciences, which is planning to use IV AAV9 gene therapy to treat Duchenne muscular dystrophy. New animal study raises concerns about high-dose gene therapy 2018-01-31T05:00:00Z Until the past decade, most psychiatric-genetics research used approaches that were successful in the 1980s and 1990s for rare disorders such as Huntington’s disease, cystic fibrosis and muscular dystrophies. Pamela Sklar (1959–2017) 2018-01-29T05:00:00Z It’s a genetic defect that causes a form of retinal dystrophy. Medical Miracles From FDA Inefficiency 2018-01-26T05:00:00Z Among the most common are cystic fibrosis, sickle cell anaemia and muscular dystrophy. Gene editing – and what it really means to rewrite the code of life 2018-01-15T05:00:00Z Morgan Hoffmann is playing for the first time since revealing in The Players Tribune that he has muscular dystrophy. Thomas returns at Sony Open and scene of scoring records 2018-01-10T05:00:00Z Muscular dystrophy actually refers to a group of more than 30 genetic diseases that cause progressive weakness and degeneration of skeletal muscles. Morgan Hoffmann returns to the PGA Tour happy and grateful after revealing his MS battle - Golf Digest 2018-01-09T05:00:00Z In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. US drug firm offers cure for blindness – at $425,000 an eye 2018-01-03T05:00:00Z Top of Collins’ list: muscular dystrophy and sickle cell. In a milestone year, gene therapy finds a place in medicine 2017-12-28T05:00:00Z The condition, retinal dystrophy, often manifests itself in young children and affects up to 3,000 Americans, Spark said. FDA Blesses Blindness Treatment That Could Cost $1 Million 2017-12-19T05:00:00Z Officials say Louisiana’s law will eventually get the drug to people with cancer, a severe form of cerebral palsy, seizure disorders, epilepsy, muscular dystrophy and other diseases. Doctors slowly applying for medical marijuana permit 2017-12-16T05:00:00Z He said the characteristics of his type of muscular dystrophy - facioscapulohumeral - are atrophy of the chest, back, neck, arms and sometimes the legs. Hoffmann says he’s playing PGA Tour with muscular dystrophy 2017-12-05T05:00:00Z Morgan Hoffmann revealed he was diagnosed with muscular dystrophy last year. Tiger Woods’ “Bright future,” Rickie Fowler’s successful Bahamas trip, and the Tiger T-shirt every fan needs for Christmas - Golf Digest 2017-12-05T05:00:00Z Morgan Hoffmann recently wrapped up a fifth full season on the PGA Tour, but it was the first in which he knew he was playing with muscular dystrophy. Morgan Hoffmann reveals he's been playing on the PGA Tour with muscular dystrophy - Golf Digest 2017-12-04T05:00:00Z A clinical trial that began in 2012 tested Luxturna in 20 patients with retinal dystrophy and compared them with nine patients who didn’t get the therapy; their average age was 15. FDA Blesses Blindness Treatment That Could Cost $1 Million 2017-12-19T05:00:00Z We don't hear of successful screening programmes for newborns, or emerging gene or cancer therapies such as those for spinal muscular atrophy or Duchenne muscular dystrophy, sickle-cell disease or leukaemia. Bioethics: Justice in genomics 2017-11-14T05:00:00Z Hoffman says he plans to start raising money and awareness of muscular dystrophy. Hoffmann says he’s playing PGA Tour with muscular dystrophy 2017-12-05T05:00:00Z Mutation in transforming growth factor beta induced protein associated with granular corneal dystrophy type 1 reduces the proteolytic susceptibility through local structural stabilization. Structure of phycobilisome from the red alga Griffithsia pacifica 2017-10-17T04:00:00Z The FDA on Wednesday rejected an experimental drug to treat muscular dystrophy. OxyContin maker faces federal investigation over claim on efficacy 2017-10-25T04:00:00Z Louisiana’s law will eventually get the drug to people with cancer, a severe form of cerebral palsy, seizure disorders, epilepsy, muscular dystrophy and other diseases. APNewsBreak: Few Louisiana doctors seek marijuana permit 2017-10-11T04:00:00Z Tests revealed that muscular dystrophy had first surfaced in an attack on Doug’s Achilles’ tendon. Making an MLB Manager 2017-09-14T04:00:00Z “Even though the type of muscular dystrophy that I have doesn’t pose an immediate threat to my life, there is a good chance that it will shorten it,” Hoffman wrote. Hoffmann says he’s playing PGA Tour with muscular dystrophy 2017-12-05T05:00:00Z Ms. Alfonzo contacted Freedom Concepts, a Canadian company that builds adaptive bicycles for children with cerebral palsy, muscular dystrophy, spina bifida and other disabilities. A Bike for Every Kid 2017-09-07T04:00:00Z Lewis’ muscular dystrophy telethons were a Labor Day staple for more than 40 years. Celebrities, loved ones remember comedian Jerry Lewis 2017-09-05T04:00:00Z The Douglas County Sheriff’s Office use of force policy prohibits police from using Tasers on people with “known neuromuscular disorders such as multiple sclerosis, muscular dystrophy or epilepsy.” Special Report: A 911 plea for help, a Taser shot and the toll of stun guns 2017-08-23T04:00:00Z Lewis was famous around the world for his partnership with Dean Martin, his fund-raising for muscular dystrophy and his numerous hit comedies. Jerry Lewis: Will his infamous 'lost' film ever be seen? - BBC News 2017-08-21T04:00:00Z They did not speak to each other for 20 years, until a mutual friend, Frank Sinatra, prodded them to appear together on Mr. Lewis’s muscular dystrophy telethon. Jerry Lewis, comedy king and master of slapstick, dies at 91 2017-08-20T04:00:00Z Over the years Lewis was criticized for allegedly exploiting young people with muscular dystrophy. Lewis’ MDA telethons were full of emotion, star appearances 2017-08-20T04:00:00Z In his case, this included muscular dystrophy, cerebral palsy, severe nearsightedness, hydrocephalus and intellectual disability. Gene Editing Might Mean My Brother Would’ve Never Existed 2017-08-09T04:00:00Z The Douglas County Sheriff’s Office use of force policy prohibits police from using Tasers on people with “known neuromuscular disorders such as multiple sclerosis, muscular dystrophy or epilepsy.” Special Report: A 911 plea for help, a Taser shot and the toll of stun guns 2017-08-23T04:00:00Z Congenital muscular dystrophy is present at birth or before age 2; the most pronounced symptom is floppiness or a lack of muscle tone. Why were a little girl’s muscles as weak as noodles? The answer was unexpected. 2017-08-04T04:00:00Z Mr. Lewis remained a television star as host of muscular dystrophy telethons, for which the Academy of Motion Picture Arts and Sciences recognized the actor with its Jean Hersholt Humanitarian Award in 2009. Jerry Lewis, comedy king and master of slapstick, dies at 91 2017-08-20T04:00:00Z Muscular dystrophy is a group of genetic diseases characterized by progressive weakness and degeneration of muscles that control movement. Lewis’ MDA telethons were full of emotion, star appearances 2017-08-20T04:00:00Z In emails released by the FDA this week, an FDA official harshly rebuked the top agency official who drove the controversial decision to approve a drug to treat a form of muscular dystrophy. FDA Emails Offer Rare Look Into Internal Battle Over a Drug’s Approval 2017-08-03T04:00:00Z Researchers have so far identified more than 120 LINE-1 gene insertions, resulting in diseases ranging from muscular dystrophy to cystic fibrosis5. Medicine's Movable Feast: What Jumping Genes Can Teach Us about Treating Disease 2017-07-26T04:00:00Z About 40 families from around the country were meeting with researchers as part of a study of congenital muscular dystrophy. Why were a little girl’s muscles as weak as noodles? The answer was unexpected. 2017-08-04T04:00:00Z The company hoped to sell the drug, aimed at combating a rare form of muscular dystrophy that afflicts boys, for $300,000 a year per patient. California Inc.: A flashy event for the world's most famous movie theater 2017-05-22T04:00:00Z The most severe and most common childhood form is Duchenne muscular dystrophy, which occurs in about one in every 3,500 male births. Lewis’ MDA telethons were full of emotion, star appearances 2017-08-20T04:00:00Z Jennifer McNary, a stay-at-home mother, was desperate to find a medicine that might spare her two sons an early death from a rare form of muscular dystrophy. How the FDA Approved a $300,000-a-Year Drug Its Own Experts Didn’t Believe Worked 2017-05-18T04:00:00Z Summit is now running phase II clinical tests of a small-molecule drug aimed at boosting protein production to treat Duchenne muscular dystrophy, a rare genetic disease. Therapeutic developments: Masters of medicine : Nature : Nature Research 2017-05-16T04:00:00Z Like her, I was born with granular dystrophy, a rare eye condition that occasionally causes “eruptions” and leaves scar tissue on my corneas. My Mom’s Vision, My Inheritance 2017-05-11T04:00:00Z How unfair that twin boys with muscular dystrophy may be denied treatment with a drug they tested in a clinical trial because their insurance company won’t pay for it. Editorials from around New York 2017-05-10T04:00:00Z Dr. Rowland founded a center at Columbia for research and treatment of A.L.S. and helped to found a center for muscular dystrophy. Lewis Rowland, Leading Neurologist on Nerve and Muscle Diseases, Dies at 91 2017-03-23T04:00:00Z Court documents say the teen had muscular dystrophy and had been bedridden for two years. Disabled teen dies from bedsore infection, 3 charged 2017-02-16T05:00:00Z For example, a gene editing therapy that boosts the muscles of patients with muscular dystrophy could perhaps be given to healthy people to give them superhuman strength. Major report prepares ground for genetic modification of human embryos 2017-02-14T05:00:00Z All children and families with Duchenne muscular dystrophy deserve access to affordable necessary care. Opinion | The pharmaceutical industry’s greed, seen as an act of generosity 2017-02-13T05:00:00Z Muscular dystrophies or skin disorders where patients lack a key structural protein, for example, are a long shot. This mysterious $2 billion biotech is revealing the secrets behind its new drugs and vaccines 2017-02-01T05:00:00Z Dr. Rowland was a prolific researcher and writer, with nearly 500 published scientific articles that focused on devastating neuromuscular diseases, including muscular dystrophy, myasthenia gravis and many rare syndromes. Lewis Rowland, Leading Neurologist on Nerve and Muscle Diseases, Dies at 91 2017-03-23T04:00:00Z His father, a former state government minister and federal government lawmaker, was recovering from open heart surgery and his mother requires 24-hour care for muscular dystrophy. Leader of Australia's most populous state quits 2017-01-18T05:00:00Z In separate research published in Nature Communications on Wednesday, scientists at the University of Washington in Seattle used gene editing to rewrite faulty genes responsible for Duchenne muscular dystrophy in adult mice. Major report prepares ground for genetic modification of human embryos 2017-02-14T05:00:00Z The youngest of three children, Yip was born with muscular dystrophy, a genetic disorder that slowly breaks down the muscles. Yip Pin Xiu: Singapore's all-conquering Paralympian - BBC News 2017-01-13T05:00:00Z Scanning the DNA of patients with Duchenne muscular dystrophy, for example, scientists have identified telltale glitches in the gene that encodes dystrophin, a protein critical to muscle stability. A Family’s Shared Defect Sheds Light on the Human Genome 2017-01-09T05:00:00Z Here’s an idea: Update part of the approval process that was patient zero for distorting data on a drug for Duchenne muscular dystrophy. The FDA’s Rigged Drug Committees 2016-12-31T05:00:00Z When you look back at the controversy over the Duchenne muscular dystrophy drug, do you have any second thoughts about the way that turned out? The head of the FDA defends the importance of drug effectiveness 2016-12-29T05:00:00Z This year produced no shortage of surprises, and a rare happy one was Food and Drug Administration approval of a muscular dystrophy drug that had been singled out for destruction by agency staffers. The FDA Empire Strikes Back 2016-12-23T05:00:00Z Ms. Estrellado, 32, has muscular dystrophy, a chronic disease in which muscles weaken and waste over time until they no longer work at all. Dating With a Disability 2016-12-08T05:00:00Z The various Crispr companies, some of them founded by scientists at UC Berkeley and the Broad Institute, are hoping to develop treatments for diseases like hemophilia, cystic fibrosis and muscular dystrophy, among others. Rival Research Teams Head to Court 2016-12-05T05:00:00Z At the time, insurance companies could still deny you coverage based on a pre-existing condition – in his case, a chronic heart condition caused by the muscular dystrophy he was diagnosed with at age three. Dismantling the ACA would be catastrophic for people like my partner | Annamarya Scaccia 2016-11-14T05:00:00Z Mr. Berlin was in his early 20s when his vision began to deteriorate, the result of cone-rod dystrophy, a degenerative condition diagnosed when he was 7. He Went Blind, Then Became a Distance Runner 2016-11-07T05:00:00Z The treatment slows the decline in patients with Duchenne muscular dystrophy, and 10 of 12 boys in a trial still walk after four years of treatment. The FDA Empire Strikes Back 2016-12-23T05:00:00Z Ally Bruener, 28, is a standup comedian with congenital muscular dystrophy. Dating With a Disability 2016-12-08T05:00:00Z Last month, the F.D.A. approved a drug to treat Duchenne muscular dystrophy even though its own scientists concluded that there was no convincing evidence the drug was effective, the usual standard for approval. Medical Charities Once Advised on Coping With a Disease. Now They Try to Cure It. 2016-10-31T04:00:00Z Diagnostic tests showed he had abnormally high liver enzymes, a symptom of muscular dystrophy. Dismantling the ACA would be catastrophic for people like my partner | Annamarya Scaccia 2016-11-14T05:00:00Z The campaign released a television ad Thursday featuring Bryce, a young man with muscular dystrophy, watching a clip of Trump mocking a disabled reporter. Clinton proposes $500 million federal program to combat bullying 2016-10-27T04:00:00Z Even after presenting in his early 20s with cone dystrophy, a rare disorder that weakened his vision, Walby won a national championship in his weight class. Paralympic Athletes’ Least Favorite Word: Inspiration 2016-09-17T04:00:00Z For Ms. Estrellado, a singer-songwriter, the worst part about having muscular dystrophy is knowing that many people don’t see her as a prospective romantic partner to date, to marry and to have children with someday. Dating With a Disability 2016-12-08T05:00:00Z In his research, he determined the chromosomal locations for more than a dozen other diseases, including Parkinson’s, Lou Gehrig’s and muscular dystrophy. Allen Roses, Who Upset Common Wisdom on Cause of Alzheimer’s, Dies at 73 2016-10-05T04:00:00Z Food and Drug Administration to grant provisional approval for eteplirsen, a new drug to treat Duchenne muscular dystrophy, is cause for celebration. Playing God in Secret 2016-10-05T04:00:00Z The different Crispr companies are hoping to develop treatments for diseases like hemophilia, cystic fibrosis and muscular dystrophy, among others. Breakthrough Gene Technology Attracts Investors Amid Patent Dispute 2016-09-22T04:00:00Z Duchenne's muscular dystrophy is a rare disease, affecting about 1 of every 3,600 boys worldwide and usually causing death by age 25, according to the National Institutes of Health. FDA Oks 1st Muscular Dystrophy Drug; Awaits Proof It Works 2016-09-19T04:00:00Z Emilie McCauley, 24, doesn’t use a wheelchair, and when people meet her, it’s not apparent that she has muscular dystrophy. Dating With a Disability 2016-12-08T05:00:00Z In April, a meeting of FDA advisers heard scientific presentations — including a highly critical review by FDA analysts — as well as passionate testimony from patients with Duchenne muscular dystrophy and their advocates. US government approves controversial drug for muscular dystrophy 2016-09-19T04:00:00Z Duchenne muscular dystrophy is a rare disease affecting mostly boys; almost all use wheelchairs by their teens and are dead by their 20s. Playing God in Secret 2016-10-05T04:00:00Z The Food and Drug Administration on Monday approved a drug for muscular dystrophy after months of delay and bureaucratic infighting. The Boys Who Beat the FDA 2016-09-19T04:00:00Z After months of advocacy and speculation, the Food and Drug Administration today granted accelerated approval to the first treatment for a rare form of muscular dystrophy. FDA grants accelerated approval to controversial muscular dystrophy drug 2016-09-19T04:00:00Z He wanted to learn about muscular dystrophy and how Ms. McCauley’s other health conditions affected her body, and offered emotional support. Dating With a Disability 2016-12-08T05:00:00Z Duchenne muscular dystrophy is caused by mutations in the dystrophin protein that protects muscle cells from the stress of repeated contraction and relaxation. US government approves controversial drug for muscular dystrophy 2016-09-19T04:00:00Z Food and Drug Administration on Monday granted preliminary approval to the first drug for a rare form of muscular dystrophy. FDA OKs 1st Drug to Treat Duchenne Muscular Dystrophy 2016-09-19T04:00:00Z The therapy is the first for Duchenne muscular dystrophy patients, typically boys who lose the ability to walk around age 12 before heart or respiratory failure in their 20s. The Boys Who Beat the FDA 2016-09-19T04:00:00Z DMD, the most common form of muscular dystrophy, is a rare genetic condition that leads to ongoing muscular deterioration and weakness. First Drug Approved for Duchenne Muscular Dystrophy 2016-09-19T04:00:00Z Duchenne is the most common type of muscular dystrophy, and typically affects boys. Controversial Drug OKd for Duchenne Muscular Dystrophy 2016-09-19T04:00:00Z The Food and Drug Administration said it cleared Sarepta Therapeutics' injection for a rare form of Duchenne muscular dystrophy, a deadly inherited disease that affects boys. FDA tentatively approves first drug for muscular dystrophy 2016-09-19T04:00:00Z National Institutes of Health provides more information on Duchenne muscular dystrophy. FDA OKs 1st Drug to Treat Duchenne Muscular Dystrophy 2016-09-19T04:00:00Z Marijuana is also approved in New Jersey to treat multiple sclerosis, terminal cancer and muscular dystrophy, among other medical diseases. Christie signs bill approving marijuana for PTSD treatment 2016-09-14T04:00:00Z Conditions such as muscular dystrophy, spina bifida and tetraplegia will fall under this classification. Rio Paralympics 2016: A-Z of Paralympic sport classification 2016-09-05T04:00:00Z The Food and Drug Administration on Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and devastating genetic disease that causes patients to lose control of their muscles. Controversial Drug OKd for Duchenne Muscular Dystrophy 2016-09-19T04:00:00Z But the event had critics - notably people with muscular dystrophy who said they were being made objects of pity. Muscular Dystrophy Association seeks post-telethon identity 2016-09-01T04:00:00Z But the event had critics — notably people with muscular dystrophy who said they were being made objects of pity. Muscular Dystrophy Association seeks post-telethon identity 2016-09-01T04:00:00Z So it's no surprise that they get diseases, from cancer to arthritis to muscular dystrophy, that seem to mimic ours. Can clinical trials on dogs and cats help people? 2016-08-11T04:00:00Z And, to name just a few hidden disabilities: multiple sclerosis, muscular dystrophy, asthma, arthritis, tendinitis, heart conditions, recent illness or surgery … Elevator no place to pass judgment 2016-07-16T04:00:00Z They said these results were especially evident among patients with Fuchs endothelial dystrophy -- a condition affecting a thin layer of cells that line the back of the cornea. Cornea Transplants Riskier for Women When They Come From Men? 2016-07-15T04:00:00Z Last week we asked the Food and Drug Administration what’s holding up a first-in-class treatment for boys with a rare and lethal form of muscular dystrophy. Boys, Not Statistics 2016-07-07T04:00:00Z Mark said most with muscular dystrophy end up in a wheelchair by age 12. Muscular dystrophy won’t keep Parrish twins from racing 2016-07-03T04:00:00Z Marijuana is currently approved in New Jersey to treat multiple sclerosis, terminal cancer and muscular dystrophy, among others. Lawmakers weigh approving marijuana for PTSD treatment 2016-06-16T04:00:00Z Hundreds of inherited disorders such as cystic fibrosis, muscular dystrophy and many types of blindness are caused by faulty genes. Gene therapy drug approval granted to GSK - BBC News 2016-05-27T04:00:00Z But a review by FDA scientists said there was virtually no evidence the drug was effective in treating muscular dystrophy. Health Highlights: May 27, 2016 2016-05-27T04:00:00Z SB271 would add, among other ailments: HIV and AIDS, cachexia, seizure disorders, epilepsy, Crohn’s syndrome, muscular dystrophy and multiple sclerosis. Louisiana editorial roundup 2016-05-25T04:00:00Z Mills’ proposal would add seizure disorders, HIV, epilepsy, muscular dystrophy, multiple sclerosis and other diseases to the list. House votes to expand Louisiana medical marijuana program 2016-05-11T04:00:00Z Mercado, who was diagnosed with spastic muscular dystrophy as a teen, has helped change that reality. Jillian Mercado made it as a model with a disability. Here’s what she wants next. 2016-04-28T04:00:00Z Drug developer Sarepta Therapeutics plunged after a Food and Drug Administration panel said its muscular dystrophy drug eteplirsen shouldn’t be approved. US stocks mostly trade higher as energy sector rises 2016-04-26T04:00:00Z Eteplirsen is an injectable drug intended to treat a subset of patients with Duchenne muscular dystrophy, a fatal genetic disease that causes increasing muscle weakness and eventually the loss of basic movement. Muscular dystrophy drug falls short at high-stakes meeting 2016-04-25T04:00:00Z An emotionally damaged man acts as caregiver to an 18-year-old with muscular dystrophy as they embark on a road trip. Every single movie coming out this summer 2016-04-24T04:00:00Z Mills wants to add seizure disorders, HIV, epilepsy, severe muscle spasms, muscular dystrophy, multiple sclerosis and other diseases to the list. On second try, Senate backs medical marijuana expansion bill 2016-04-20T04:00:00Z A drug for treating children aged five and over with Duchenne muscular dystrophy will be available for around 50 youngsters through NHS England. Green light for Duchenne muscular dystrophy drug - BBC News 2016-04-15T04:00:00Z In 2004, Emma got a permanent weekend job working in Manhattan for a woman with muscular dystrophy, and she learned to dissociate herself from the most unpleasant aspects of the job. Mother for Hire 2016-04-11T04:00:00Z But because of how the trial was structured, the FDA said, comparing study participants to typical muscular dystrophy patients would be an “apples to oranges” comparison. Muscular dystrophy drug falls short at high-stakes meeting 2016-04-25T04:00:00Z Duchenne muscular dystrophy is a sex-linked disease that causes muscle degeneration starting around 2 years old leading to profound weakness, a need for braces and wheelchairs by age 10, and eventual paralysis. Indiana’s new abortion law won’t save babies. It will only make my patients suffer. 2016-04-01T04:00:00Z Mercado, who was diagnosed with spastic muscular dystrophy when she was a child, grew up loving fashion. Beyoncé Commissions Model With Muscular Dystrophy to Promote New Campaign 2016-03-18T04:00:00Z When I was diagnosed with muscular dystrophy 10 years ago, at the age of 29, my life was pretty normal. Are disabled people's sex lives being ignored? - BBC News 2016-02-19T05:00:00Z Hopes are dimming that the drug industry will be able to quickly deliver a treatment for one of the most keenly researched diseases of recent years—an inherited muscle-wasting disorder called Duchenne muscular dystrophy. Muscular Dystrophy Drugs Face New FDA Questions 2016-01-21T05:00:00Z Families and patients affected by muscular dystrophy packed the FDA committee meeting, often applauding comments from Sarepta scientists while openly rebuking FDA regulators. Muscular dystrophy drug falls short at high-stakes meeting 2016-04-25T04:00:00Z Scientists edited the DNA of mice to treat muscular dystrophy. Why Saudi Arabia Is Heating Up Its Cold War With Iran 2016-01-05T05:00:00Z Duchenne muscular dystrophy is a progressive muscle-wasting disease that affects boys, putting them in wheelchairs by age 10, followed by an early death from heart failure or breathing difficulties. Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find 2015-12-31T05:00:00Z And some people have been at this game a long time, if you think about how much money has been raised for cystic fibrosis and muscular dystrophy, and we don’t have a cure.” Parents of Children With Rare Diseases Find Hope in For-Profit Companies 2015-12-25T05:00:00Z There are an estimated 28 trials or studies for potential therapies involving a drug for Duchenne or closely related Becker muscular dystrophy. Muscular Dystrophy Drugs Face New FDA Questions 2016-01-21T05:00:00Z The company focuses on advance treatments and finding a cure for Duchenne muscular dystrophy patients. Company pays $2M to research muscular dystrophy treatment 2015-12-15T05:00:00Z The couple’s two sons, who had muscular dystrophy, died in their 20s. Henry M. Rowan, Industrialist Who Gave $100 Million Gift to University, Dies at 92 2015-12-13T05:00:00Z This is what happens in a milder disease known as Becker muscular dystrophy, in which mutations cause instructions from a few exons to be skipped during the protein-making process. Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find 2015-12-31T05:00:00Z The prognosis for people who have the disease, considered a severe form of muscular dystrophy, is to need to use a wheelchair by the time they are teenagers. Parents of Children With Rare Diseases Find Hope in For-Profit Companies 2015-12-25T05:00:00Z As the program exists today, patients need a doctor or other caregiver to certify they have one of nine serious conditions, such as cancer, epilepsy, muscular dystrophy or Crohn’s disease. Excerpts from recent Minnesota editorials 2015-12-07T05:00:00Z They're developing treatments for muscular dystrophy, sickle cell disease, cancer and HIV. Scientists, ethicists begin tackling questions about how to use powerful new gene-editing tool 2015-12-02T05:00:00Z They are developing potential treatments for muscular dystrophy, sickle-cell disease and cancer. Scientists debate ethics of human gene editing at international summit 2015-12-01T05:00:00Z As revealed on a recent episode of “The Doctors” TV show, Ms. Guzman suffers from muscular dystrophy, a debilitation disorder that causes the restricting and tightening of muscles and a lifetime — often shortened — of pain. Amber Guzman on her life as a ‘living doll’ and muscular dystrophy 2015-11-29T05:00:00Z At a parliament session this week, Hazan was heard belittling Karin Elharar, an opposition lawmaker who suffers from muscular dystrophy and uses a wheelchair. Controversial Israeli lawmaker once again under fire after mocking disabled colleague 2015-11-27T05:00:00Z The bill would include additional students on the autism spectrum, students with muscular dystrophy, and three- and four-year-old children who meet certain criteria. Florida may expand help for intellectually disabled children 2015-11-18T05:00:00Z Scientists hope that by studying Ringo’s mutation — which has never before been linked to muscular dystrophy — they can find new treatments for the disease. Puppy bred to have muscular dystrophy saved by surprise mutation 2015-11-11T05:00:00Z If this new drug worked, it would transform Austin and Max’s disease into a milder form of muscular dystrophy called Becker’s. Dying Should Have 'Right to Try' New Treatments 2015-11-10T05:00:00Z But what really carried me into it was my illness, muscular dystrophy, which is affecting my body and causing me to lose my muscles, which is making it harder for me to move. Amber Guzman on her life as a ‘living doll’ and muscular dystrophy 2015-11-29T05:00:00Z This test has been used to predict the survival rates of lung transplant candidates, to measure the progression of muscular dystrophy, and to assess overall cardiovascular fitness. Mobile data: Made to measure : Nature : Nature Publishing Group 2015-11-03T05:00:00Z Researchers are building stronger immune cells, fighting muscular dystrophy in mice and growing human-like organs in pigs for possible transplant. HEALTHBEAT: Gene-editing research seeks to balance ethics with potential for fighting disease 2015-10-09T04:00:00Z Researchers bred him and his littermates to inherit a gene mutation that causes severe muscular dystrophy. Puppy bred to have muscular dystrophy saved by surprise mutation 2015-11-11T05:00:00Z The treatment was tested in patients with RPE65-mediated inherited retinal dystrophies. Spark Therapeutics up after detailing eye treatment study 2015-10-05T04:00:00Z A late-stage clinical trial showed that people with inherited retinal dystrophies were better able to move around in low light after receiving the treatment, according to developer Spark Therapies, The New York Times reported. Health Highlights: Oct. 5, 2015 2015-10-05T04:00:00Z Patrick McClellan, who suffers crippling muscle spasms from muscular dystrophy, told The Associated Press that his monthly tab for oil for vaporizer pens - one of the legal state-sanctioned treatments - runs $264. Some Minnesota marijuana patients opting to buy illegally 2015-09-20T04:00:00Z Patrick McClellan, who suffers crippling muscle spasms from muscular dystrophy, told The Associated Press that his monthly tab for oil for vaporizer pens — one of the legal state-sanctioned treatments — runs $264. Stung by costs, some of Minnesota's medical marijuana patients back to buying on streets 2015-09-20T04:00:00Z When the researchers mimicked this trait in zebrafish lacking dystrophin, it also protected the fish from muscle tearing and other symptoms of muscular dystrophy. Puppy bred to have muscular dystrophy saved by surprise mutation 2015-11-11T05:00:00Z Mr. Lewis launched his solo career in 1956, and became known for his philanthropy, mostly notably hosting an annual Labor Day telethon to raise money for muscular dystrophy research. Jerry Lewis trove to be preserved by Library of Congress 2015-09-14T04:00:00Z The exhibition—being presented by Stardust Monte Carlo, a longtime Monaco-based luxury jeweler—supports the Prince Albert II of Monaco Foundation, dedicated to environmental conservation; and Only Watch, a biennial auction benefitting muscular dystrophy research. 75-Carat Yellow Diamond Leads Jacob & Co. Exhibit in Monaco 2015-08-03T04:00:00Z In my primary school in Anchorage, Alaska, some of the children used to shove snow down the jacket of a boy who had muscular dystrophy, because his physical disability prevented him from protecting himself. Do people become more prejudiced as they grow older? - BBC News 2015-07-16T04:00:00Z Root, a Navy veteran, was diagnosed with muscular dystrophy in the middle of her military career, and had to retire. Disabled veterans compete in Valor Games at Duke Univ. 2015-06-01T04:00:00Z The MDA aims to raise awareness and improve the lives of people suffering from muscular dystrophy, a rare muscle-wasting genetic disease, amyotrophic lateral sclerosis and other related diseases. Muscular dystrophy telethon, long run by Jerry Lewis, ends on ABC 2015-05-01T04:00:00Z There was testing for muscular dystrophy, Parkinson’s disease, cerebral palsy. Belle of the ball was ready for her big night 2015-04-27T04:00:00Z Doctors said that even if he survived, he would likely face complications that include blindness, deafness or muscular dystrophy. Wisconsin boy, 5, born size of dollar bill beats odds 2015-04-20T04:00:00Z Lobu, almost certainly has inherited muscular dystrophy, a genetic condition that causes the muscles to gradually weaken, leading eventually to total disability and, in some cases, death. The 'cursed' disabled boy and the people of the sea 2015-04-16T04:00:00Z At our age, past the prime of life, we are given to sinew and sag, and Larry bears, with his trademark stoicism, the further affliction of a late-onset muscular dystrophy. Sally Mann’s Exposure 2015-04-16T04:00:00Z The MDA said it will be investing in digital and mobile platforms to engage with the public, and will continue sharing stories of families living with muscular dystrophy on Labor Day. Muscular dystrophy telethon, long run by Jerry Lewis, ends on ABC 2015-05-01T04:00:00Z In Newport Beach, California, Debra Miller devoted her life to finding a cure for Duchenne muscular dystrophy, which her son suffers from. The Top Three Most Difficult Decisions for Philanthropists 2015-03-18T04:00:00Z Jordan is battling a terminal form of muscular dystrophy called Duchenne, and he’s not expected to live past the age of 20. 5-year-old firefighter lobbies for right-to-try bill 2015-03-04T05:00:00Z There is no cure for muscular dystrophy, steps can only be taken to alleviate the most painful symptoms. The 'cursed' disabled boy and the people of the sea 2015-04-16T04:00:00Z This is a "monumental moment" in efforts to fight muscular dystrophy, according to Robert Meadowcroft, chief executive of the Muscular Dystrophy Campaign. Health Highlights: Feb. 25, 2015 2015-02-25T05:00:00Z Patients with Duchenne muscular dystrophy lose the ability to walk between the ages of 7 and 13, and die in their teens or 20s. Researchers Measure Scope of Muscular Dystrophies That Strike Boys 2015-02-20T05:00:00Z Defects in the mitochondria — energy-producing structures outside a cell’s nucleus — can result in a range of complications, including muscular dystrophy and heart, kidney and liver failure. Britain Nears Approval of Fertilization Technique That Combines DNA of Three People 2015-02-03T05:00:00Z Muscular dystrophy is a group of diseases that cause progressive weakness and a loss of muscle mass. Welding class in Texas City offers education in life 2015-01-27T05:00:00Z The 38-year-old Emery resident, who’s been diagnosed with reflex sympathetic dystrophy, leads a group working on a bill that would allow for whole-plant remedies including oils, edibles and creams. Signs advocating for medical marijuana coming to SD 2015-01-02T05:00:00Z Journalist Nikki Fox, who serves as a disability correspondent for BBC News, was born with muscular dystrophy and hadn’t taken a step on her own in ten years. Watch This Woman Take Her First Steps in Ten Years 2014-12-18T05:00:00Z "There were always some rather crude estimates of how common these muscular dystrophies are," study corresponding author Paul Romitti, an epidemiologist at the University of Iowa, said in a university news release. Researchers Measure Scope of Muscular Dystrophies That Strike Boys 2015-02-20T05:00:00Z Participants typically include people with autism, cerebral palsy, speech impairment, emotional, behavioral or mental health issues, muscular dystrophy, visual and hearing impairment, Down syndrome and multiple sclerosis. Horses help provide healing in Howell area 2014-12-07T05:00:00Z Two biotech companies, Sarepta and Prosensa Therapeutics, are hoping to get FDA approval based on measurements that show their drugs improve levels of dystrophin, the protein that is missing in muscular dystrophy, in some patients. A Day Of Reckoning Approaches For Merck And Its Critics 2014-11-13T05:00:00Z The other day, Leffler found a piece of paper in her bathroom with a question written in Aidan’s neat block letters: “Does muscular dystrophy make you die sooner?” Moms, Regulators, Biotech Startups, and the Battle Over a Potentially Life-Saving Drug 2014-10-30T04:00:00Z Additional requirements placed on the drug developer appear to be without regard to the pleas of the Duchenne muscular dystrophy community to hurry up. MedidataVoice: Rare Disease Patient Voices Bring Change To The Clinical Trials Process 2014-09-25T04:00:00Z He says other roles he’s taken on include that of a young man with muscular dystrophy and another where he was “a bit mentally unstable.” Actor RJ Mitte sees cerebral palsy as an asset 2014-09-10T04:00:00Z There’s a grim understanding between the two teens: A terminal form of muscular dystrophy is slowly consuming Colton’s body, paralyzing all but his forearms. Teens’ friendship blossoms amid terminal disease 2014-08-22T04:00:00Z An estimated one million Americans live with the disease, which is more than those diagnosed with multiple sclerosis, muscular dystrophy and Lou Gehrig’s disease combined, according to the foundation. Boxing eases Dighton man’s Parkinson’s symptoms 2014-08-24T04:00:00Z If proven safe and effective, the drugs would turn Duchenne into a less devastating form of muscular dystrophy. Moms, Regulators, Biotech Startups, and the Battle Over a Potentially Life-Saving Drug 2014-10-30T04:00:00Z In a recent case, muscular dystrophy patient advocates developed a Risk vs. MedidataVoice: Rare Disease Patient Voices Bring Change To The Clinical Trials Process 2014-09-25T04:00:00Z A physiologist at the University of Buffalo is trying to heal muscular dystrophy patients with a compound in the venom of a South American spider. The case for spider conservation: They keep pests from devouring humans’ food supply. Ahbid Choudry, 34, was diagnosed with muscular dystrophy when he was 13, a condition which left him unable to walk and with a weakened immune system. Health warning over Ramadan fasting 2014-07-10T04:00:00Z Michael was 5 when he was diagnosed with Duchenne muscular dystrophy, a genetic disorder that causes progressive muscle degeneration and weakness. Texas man fights to get off the road to paralysis 2014-07-06T04:00:00Z Writing in Nature, Cattaneo and Corbellini highlight the case of the Stamina Foundation, a private organization in Italy that offers what it describes as 'stem-cell therapy' for Parkinson's disease, muscular dystrophy and spinal muscular atrophy. Secret publishing deals exposed : Nature : Nature Publishing Group 2014-06-24T04:00:00Z The Movement, a DC Comics series, focusing on a group of diverse teenagers, features the character of Vengeance Moth, a 19-year-old woman who uses a wheelchair, because of her muscular dystrophy. Superheroes with a difference 2014-06-24T04:00:00Z Recently two patients with muscular dystrophy, both in wheelchairs, married in the courtyard outside. Life Goes On at Long-Term Acute Care Hospitals 2014-06-23T04:00:00Z MacDougall graduated from the Annapolis college last month at age 19 - she skipped high school - despite suffering from spinal muscular atrophy, a type of muscular dystrophy. St. John’s grad seeks funds for master’s in UK 2014-06-14T04:00:00Z Mutations in single genes cause many other diseases such as Tay-Sachs disease or some types of muscular dystrophy; the prognoses for people born with these conditions are similarly bleak. Genetic Heroes May Be Key to Treating Debilitating Diseases 2014-05-30T04:00:00Z Tiny weights like these weren’t really available anywhere else, and Baniak told Isabella that they could be helpful for many conditions in children, such as prematurity, infant stroke, autism, cerebral palsy and muscular dystrophy. W.Va. company makes tiny weights to help kids 2014-05-26T04:00:00Z Wani, who has a biochemical engineering degree and suffers from muscular dystrophy, lives in Manchester, New Hampshire and became a US citizen in 2005. Sudan faces mounting condemnation over pregnant woman's death sentence 2014-05-25T04:00:00Z There are 2,400 children in the UK living with muscular dystrophy, but only those whose condition is caused by a particular 'nonsense mutation' - namely 200 children - are suitable to use Translarna. Duchenne drug 'nears approval' in EU 2014-05-23T04:00:00Z Overby was diagnosed with reflex sympathetic dystrophy syndrome, a rare and extremely painful nerve condition. Pain and gain: An Alabama clinic stands out amid data on Medicare payments 2014-05-11T00:22:00Z The drug, ataluren, is intended to treat a form of Duchenne muscular dystrophy which occurs due to a type of genetic mutation known as a nonsense mutation. EU committee recommends against PTC's muscular disorder drug 2014-01-24T12:54:56Z He also held fundraisers, not only to help with the cost but to raise money for CureDuchenne, a nonprofit group that funds research for a type of muscular dystrophy. Boy and dad climb tallest peak in Americas 2014-01-06T00:21:41Z His father said the judge took into account the fact that the team were raising money for muscular dystrophy research. Nine-year-old climbs Argentine peak 2013-12-28T00:53:07Z Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function. Duchenne drug 'nears approval' in EU 2014-05-23T04:00:00Z Dutch drugmaker Prosensa Holding saw its stock soar 54 percent in their U.S. market debut on June 28, a day after the FDA granted breakthrough status to the company's experimental muscular dystrophy drug. Analysis: Investment risks of fast-tracking biotech drugs become apparent 2013-12-18T06:02:14Z There are more than 60 types of muscular dystrophy genetic disorders A group of MPs is calling for a ring-fenced fund to pay for drugs for rare conditions. Call for rare conditions drugs fund 2013-09-02T23:57:34Z Mr. Segall sponsored charitable events for causes like juvenile arthritis, muscular dystrophy and the families of Sept. Family Struggles to Salvage Livelihood Damaged by Hurricane 2013-08-20T02:02:01Z IPO craze instead, was Dutch firm Prosensa Holding, a specialist in drugs to treat muscular dystrophy. Venture capital warms to European biotechs 2013-08-07T11:30:27Z The bill lists more than 30 ailments, such as cancer, muscular dystrophy and lupus. Domestic briefs: Threat prompts U.S. to temporarily close embassies in Muslim world 2013-08-02T00:24:35Z The key fact is that boys with muscular dystrophy tend to decline quickly and die in their early twenties. Innovative Muscular Dystrophy Drug Edges Closer To Market 2013-07-24T11:40:00Z Mutations in these previously described genes are responsible for the so-called X-linked recessive diseases such as hemophilia and Duchenne muscular dystrophy. 'Female' Chromosome May Leave a Mark on Male Fertility 2013-07-21T18:30:00Z It involves intervening in the fertilization process to remove faulty mitochondrial DNA, which can cause inherited conditions such as fatal heart problems, liver failure, brain disorders, blindness and muscular dystrophy. Britain plans world's first go-ahead for '3-parent' IVF babies 2013-06-28T05:58:29Z The research will include animal studies and some early clinical trials for eight diseases, including Duchenne muscular dystrophy, alcohol and nicotine addiction, and common disorders like Alzheimer's. NIH Announces Nine Projects to Repurpose Old Drugs 2013-06-18T22:35:00Z For example, scientists found the zebra fish mutation which corresponds to the mutated gene in those with muscular dystrophy, a rare genetic condition. A Point Of View: Fly, Fish, Mouse and Worm 2013-06-14T16:55:00Z One of the best-case scenarios for eteplirsen would be if the FDA would accept measures of dystrophin, a muscle protein not made in muscular dystrophy, as proof of the drug’s efficacy. Innovative Muscular Dystrophy Drug Edges Closer To Market 2013-07-24T11:40:00Z It is an illegal offshoot of gene therapy, which typically alters a person's DNA to fight diseases like muscular dystrophy and cystic fibrosis. WADA to hold meeting in China on gene doping 2013-05-03T10:34:09Z Drug companies, meanwhile, are developing a raft of new medications for diseases like muscular dystrophy and anemia that could one day be used as enhancers. News Analysis: So Long, Lance. Here Comes 21st-Century Doping. 2013-01-19T17:06:21Z Mr. Celik, 23, has Duchenne muscular dystrophy, a rapidly progressive form of the disease, which has paralyzed most of his body except for a few fingers on each hand. The Neediest Cases: Hurricane Sandy Victim, Ill, Is Restricted in New Home 2013-01-16T04:27:03Z The larger the area of so-called dystrophy, the more likely a patient was to fail the test. Dermatitis can lead to fingerprint ID failures 2012-12-28T20:26:55Z That seems to be a careful signal from the FDA that it can’t assure either Sarepta or muscular dystrophy patients of an approval. Innovative Muscular Dystrophy Drug Edges Closer To Market 2013-07-24T11:40:00Z Pig models are now being developed for other common conditions, including Alzheimer’s disease, cancer and muscular dystrophy. Pig geneticists go the whole hog 2012-11-14T18:21:06.233Z Shazier wore No. 48 on Saturday to honor a friend who, he said, died last summer from muscular dystrophy. No. 9 Ohio State 35, Penn State 23: Ohio State Beats Penn State in Battle of the Banned 2012-10-28T06:10:24Z Proceeds will be split between Kluwe’s charity, Kick for a Cure, which benefits children with Duchenne muscular dystrophy, and Minnesotans for Equality. Punter Chris Kluwe’s Voice Is Heard in Gay Marriage Debate 2012-10-19T19:52:14Z The son of a Haitian construction magnate, Oriol as a child was diagnosed with spinal muscular atrophy, a genetic disease similar to muscular dystrophy. Haiti’s government and charities work to improve access for people with handicaps 2012-10-15T20:35:00Z The drug, called eteplirsen, also appeared to restore levels of the key protein that muscular dystrophy patients lack to about half of normal levels, Sarepta said. Drug Shows Promise in Treating Type of Muscular Dystrophy 2012-10-03T14:24:42Z Those results are “incredibly promising,” says Lee Sweeney, chairman of physiology at the University of Pennsylvania’s Perelman School of Medicine, who studies muscular dystrophy and was not involved in the study. In A Dramatic Result, An Experimental Drug May Help Boys With Muscular Dystrophy 2012-10-03T11:08:52Z Drew had muscular dystrophy and got around on a motorized scooter, but, as Sarah says, he never complained about anything. The Fifth Quarter: Two dominating defenses, league races to watch and rankings 2012-10-01T10:50:45Z Nottinghamshire's Hynd, who has muscular dystrophy, said: "I'm happy with that, but I would have liked the time to be quicker." Frederiksen wins backstroke gold 2012-09-04T17:53:26Z Sweeney and other researchers hope that these sorts of gene therapies can benefit the elderly, patients suffering from muscular dystrophy and even astronauts who lose muscle mass in a zero-gravity environment. Should Fans Fret that Olympians Are Fiddling With Their Genes? 2012-07-28T11:45:03.747Z The gene that causes Duchenne muscular dystrophy was discovered in 1986, yet it is only now that drugs based on that finding are making the way to market. Drug Shows Promise in Treating Type of Muscular Dystrophy 2012-10-03T14:24:42Z The new medicine, eteplirsen, is targeted at the 13% of those boys whose muscular dystrophy is caused by a certain type of mutation. In A Dramatic Result, An Experimental Drug May Help Boys With Muscular Dystrophy 2012-10-03T11:08:52Z But there was a problem: Baughman suffers from limb girdle muscular dystrophy, which makes it hard for her walk or stand up from a seated position. Does Disneyland Discriminate Against the Disabled? 2012-07-23T12:00:34Z Morris's condition, reflex sympathetic dystrophy or RSD, involves a malfunction of the nervous system which causes severe pain and makes her extremely sensitive to injury. Paralympic cycling medal hopeful may miss Games after being hit by car 2012-07-10T13:24:54Z These findings suggest that transplantation of genetically corrected mesoangioblast-like cells generated from iPSCs from LGMD2D patients may be useful for treating this type of muscular dystrophy and perhaps other forms of muscular dystrophy as well. [Research Articles] Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy 2012-06-27T18:55:16.800Z Additional Test Results: Dr. Gilchrist ordered a muscle biopsy to look for evidence of any injury to the muscle that would suggest a form of muscular dystrophy. Well: Think Like a Doctor: A Crooked Walk 2012-04-05T04:18:37Z He suffers from muscular dystrophy, which leaves the muscles in his leg weak. 'Blade Runner' legs for sprinter 2012-03-13T05:56:58Z It’s a major health burden, more common than muscular dystrophy, Parkinson’s and multiple sclerosis put together. Surgery for Epilepsy Gains Urgency in Trial 2012-03-12T21:20:06Z Scientists are testing a daring new treatment for both macular degeneration and macular dystrophy. Stem Cells: Flexible friends 2012-02-29T18:21:27.080Z One of the annual, celebrated functions in Stamford, a city of 120,000 about 35 miles northeast of Manhattan, is a dinner benefiting muscular dystrophy. Luck and Loyalty Keep Bringing Bobby Valentine Back 2012-02-17T19:24:05Z The biopsy showed no sign of muscular dystrophy. Well: Think Like a Doctor: A Crooked Walk 2012-04-05T04:18:37Z The approach raises ethical questions, but could spare children from inheriting some rare diseases, including forms of muscular dystrophy and neurodegenerative disorders that affect around 1 in 5,000 people. UK sets sights on gene therapy in eggs 2012-01-25T18:50:18.833Z The other, who asked that her name not be used to protect her privacy, was a 51-year-old graphics designer in Los Angeles with Stargardt’s macular dystrophy, which tends to occur in younger people. Stem Cell Study May Show Advance 2012-01-23T17:57:27Z Thousands of people are diagnosed annually with illnesses clearly rooted in genes, such as Huntington’s disease, muscular dystrophy and Down syndrome. Genome Map Lets Kristal James Become a Mom Again Beating Cancer 2012-01-23T13:30:39Z Last Sunday, less than nine hours before kickoff against the New York Jets, Bowen’s mother-in-law, Muriel Johnson, died at 59 of muscular dystrophy. Redskins try to slow down Tom Brady and New England when they host Patriots on Sunday 2011-12-09T09:52:00Z Muscular dystrophy: a group of diseases in which muscles slowly deteriorate because of an inherited defect in the muscle cell. Well: Think Like a Doctor: A Crooked Walk 2012-04-05T04:18:37Z In the early morning hours Sunday, his mother-in-law, Muriel Johnson, passed away after a long bout with muscular dystrophy. Stephen Bowen wins Redskins courage award 2011-12-08T17:32:22Z Mr. Wogan, who used a wheelchair because of muscular dystrophy, died early Saturday. Flight Data Recovered From Plane in Reno Air-Race Crash 2011-09-19T01:21:01Z She was a doctoral pharmacology student who worked on a team that experimented on mice as part of research into enzymes that could have implications for the treatment of cancer, diabetes and muscular dystrophy. Family of slain Yale graduate student sues university, claims school failed to protect women 2011-09-06T22:17:20Z Muscular dystrophy put the San Diego woman, 74, in a wheelchair, but her own spirit created the Wheelchair Dancers Organization. Seniors shine at pageant 2011-09-01T19:00:06Z Lewis told a group of television critics just before the MDA announced the split that he would explain everything after the telethon, hinting that he plans to still raise money for muscular dystrophy work. Without Lewis as top pitchman, MDA asks Americans for $1 more in shortened 6-hour telethon 2011-09-04T17:27:28Z When asked what he'd have to do to be satisfied with his life, Lewis said: "Get the cure for muscular dystrophy, then I'm fine." Lewis, MDA mum on reasons for comedian's exit 2011-08-05T12:05:09Z When asked what he’d have to do to be satisfied with his life, Lewis said: “Get the cure for muscular dystrophy, then I’m fine.” Lewis, Muscular Dystrophy Association mum on why comedian leaving charity tied to telethon 2011-08-05T08:12:47Z It raised almost $59 million to fund research to find a cure for muscular dystrophy and ALS, or Lou Gehrig's disease. Jerry Lewis no longer MDA's national chairman 2011-08-04T12:46:03Z It raised almost $59 million to fund research to find a cure for muscular dystrophy and ALS, or Lou Gehrig’s disease. MDA says comedian Jerry Lewis no longer national chairman, won?t appear on next telethon 2011-08-04T04:34:49Z Duchenne muscular dystrophy affects one in every 3,500 newborn boys. 'Scalpel' hope for muscle disease 2011-07-25T03:35:31Z Amanda Muccio, 15, a ninth grader in a biology class, said she asked Ashley, also 15, whether she was embarrassed to meet boys because she had muscular dystrophy and used a wheelchair. Learning Empathy by Looking Beyond Disabilities 2011-06-22T03:51:09Z Howie never let muscular dystrophy slow him down, and he refused to let elbow surgery get in his way. Bellevue Christian trio still in the game 2011-05-21T02:39:05Z The substance - utrophin - seems to compensate for the lack of a key protein, dystrophin, which does not work properly in muscular dystrophy. Drug hope for muscular dystrophy 2011-05-05T01:10:41Z The twins were born with a rare form of muscular dystrophy. Why Entrepreneurs Should Take Sick Days 2011-05-03T04:00:00Z We all carry several hundred genes for disease: cystic fibrosis, diabetes, phenylketonuria, muscular dystrophy. Op-Ed Contributor: Unsafe at Any Dose 2011-04-30T21:14:02Z He heads a separate research unit that Pfizer opened last year to search for medications for certain distinctively gene-based rare diseases, such as muscular dystrophy and hereditary emphysema. Push to spur more drugs for deadly rare diseases 2011-04-25T22:01:06Z There were tests and eventually a diagnosis, facioscapulohumeral dystrophy, which is caused by a missing piece of DNA on chromosome 4. Nets broadcaster Carrino talks of life with MD 2011-04-13T18:27:12Z The most promising candidate, named SMT C1100, was tested in a mouse model of muscular dystrophy at three laboratories in Oxford, Italy and the US. Drug hope for muscular dystrophy 2011-05-05T01:10:41Z Austin said he pays a significant amount of money for health care, particularly the drugs his wife takes to control her muscular dystrophy. GOP governors gambling on challenge to public-employee unions 2011-02-27T05:00:00Z I had a lot of fun in those college years and learned many leadership lessons by managing events like “the world’s largest toga party” to raise money to fight muscular dystrophy. The Boss: From Software to Satellites 2011-02-26T19:02:37Z Other labs are building cell lines from patients with muscular dystrophy, Type 1 diabetes, Parkinson's disease, Huntington's disease and immune deficiencies. Stanford research team builds tiny broken heart to study lethal ailment 2011-02-10T05:36:03Z One of the most common is Duchenne muscular dystrophy, which usually affects boys. Are the Beckhams more likely to have another boy? 2011-01-10T13:03:25Z In November, ACT won FDA approval for the second human trial of human embryonic stem cells to treat people with a progressive form of blindness called Stargardt's macular dystrophy. FDA approves second Advanced Cell stem cell trial 2011-01-03T16:34:50Z Now 21, he is paralyzed as a result of Duchenne muscular dystrophy, characterized by the progressive loss of muscle strength. The Neediest Cases: Losing His Mobility, but Not His Musical Dreams 2011-01-02T04:36:44Z In November Advanced Cell Technology, of Massachusetts, won approval for the trial of a treatment for Stargardt’s macular dystrophy, a disease that destroys retinal cells in children. Embryonic stem cells: Testing science 2010-12-09T10:31:00Z Duchenne muscular dystrophy affects an estimated one in 3,500 males, according to the National Institutes of Health. Viagra may help heart effects of muscular dystrophy 2010-10-18T19:45:00Z The company has asked the FDA for permission to use it in patients with Stargardt's macular dystrophy, a genetic eye disease that progresses to blindness. First patient treated in Geron stem cell trial 2010-10-11T13:26:00Z She won gold in the team compound archery today, despite being affected by reflex sympathetic dystrophy, which causes chronic pain in her feet. Commonwealth Games 2010: Rebecca Adlington eases to 800m freestyle win 2010-10-07T19:29:00Z Mr. Lewis said the money would allow the association to continue financing worldwide research to find treatments and cures for muscular dystrophy and related diseases. Telethon Says $58.9 Million Was Raised 2010-09-07T02:11:00Z Driscoll thought fragile X was less likely than a mild form of muscular dystrophy. Mother labored to find reason for son's developmental delays 2010-08-24T04:00:00Z Trailblazer, a campaign group for people with muscular dystrophy, claims this restriction excludes most powered wheelchairs, which weigh an average of 100kg with their batteries detached. EasyJet taken to task over wheelchair policy 2010-08-22T23:05:00Z Muscular dystrophy management Reflects percentage of muscular distrophy patients age 5 or older who had a lung function test in past year or within 90 days of undergoing general anesthesia. Best Children's Hospitals 2010-11: Behind the Terms 2010-06-02T22:00:00Z Under current UK law this is not allowed, except in order to avoid gender-linked diseases, such as haemophilia and muscular dystrophy. Gender dilemma 2010-05-26T05:01:00Z Howie deals with the daily struggles of muscular dystrophy, which causes him to constantly work on building and maintaining muscle. Bellevue Christian trio not caught looking 2010-05-04T03:51:00Z Doctors at a clinic in Kingston gave her a diagnosis of muscular dystrophy. A Young Disabled Teacher Navigates a Vertical City 2010-05-01T17:49:00Z The list of illnesses to be studied in the resulting grants reveals the scope of the emerging field: cancer, Alzheimer's disease, autism, bipolar disorder, schizophrenia, asthma, kidney disease, glaucoma, muscular dystrophy and more. 2009-12-15T05:00:00Z Programs include those for brachial plexus injuries, muscular dystrophy, neurofibromatosis, pain, skeletal dysplasia, spasticity, and spina bifida. Best Children's Hospitals 2010-11: Behind the Terms 2010-06-02T22:00:00Z Marie regards the disease as a systemic dystrophy analogous to myxedema, due to a morbid condition of the pituitary body, just as myxedema is due to disease of the thyroid. Anomalies and Curiosities of Medicine There is a singular muscle shrinking and deforming disease, known as progressive muscular dystrophy, hitherto a complete and unsolved mystery. The Glands Regulating Personality |
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