| 单词 | hemophilia |
| 例句 | The economic, political, and social forces that unloosed themselves on the streets of Petrograd and launched the Russian Revolution were vastly more complex than Alexei’s hemophilia or Rasputin’s machinations. The Gene 2016-06-02T00:00:00Z Sir Urre of Hungary had long been carried from country to country—perhaps it was a sort of hemophilia—searching for the best knight who would be able to help him. The Once and Future King 1958-01-01T00:00:00Z For traits such as hemophilia, this was straightforward: one hardly needed twin studies to establish heredity. The Gene 2016-06-02T00:00:00Z The mutation in the hemophilia gene, which affects the clotting of blood, had likely arisen spontaneously in Queen Victoria at birth. The Gene 2016-06-02T00:00:00Z He worked odd jobs waiting tables and doing construction, but he’d eventually have another hemophilia attack and end up unemployed again. The Immortal Life of Henrietta Lacks 2010-02-02T00:00:00Z A blood supply tainted with HIV killed thousands of people with hemophilia in the early years of the epidemic, before tests were in place to screen for it. Review | A doctor, a parent, a dying child. A play about what medicine can and cannot do. 2018-04-12T04:00:00Z I didn’t speak Russian, and was interested mainly in military history, but I knew what the impact of hemophilia was in a family, especially when there were no transfusions. Robert K. Massie?s Latest Subject Is Catherine the Great 2011-11-09T22:40:21Z It’s hard to think of two modern presidents with a more dire case of political hemophilia. ‘Richard Nixon,’ Portrait of a Thin-Skinned, Media-Hating President 2017-03-29T04:00:00Z She helped raise funds for museums, art galleries and charities, including hemophilia research and hunger, and turned down many requests for interviews and guest appearances. Lizabeth Scott, Film Noir Siren, Dies at 92 2015-02-06T05:00:00Z The young Mr. Ailes suffered from hemophilia and was often picked on and beaten by bullies. Roger Ailes, architect of conservative TV juggernaut Fox News, is dead at 77 2017-05-18T04:00:00Z The 18-year-old died on April 8, 1990, about five years after contracting HIV, the virus that causes AIDS, through a tainted blood transfusion to treat his hemophilia. Elton John says Ryan White turned his life around 2010-04-29T01:51:00Z The success of the tests showed that engineered AsCas12f has the potential to be used for human gene therapies, such as treating hemophilia, a disease in which the blood does not clot normally. Small but mighty new gene editor 2023-09-29T04:00:00Z He died May 17, 2017, due to injuries from a fall aggravated by lifelong hemophilia. Prayer, faith were key when Gretchen Carlson took on Fox News in epic lawsuit 2023-09-19T04:00:00Z For example, Gilbert says, "People with hemophilia are unable to clot blood properly, which means that even a minor cut can have serious consequences." Blood clots kill more Americans than breast cancer or car accidents. Here's how to know the signs 2023-07-31T04:00:00Z Food and Drug Administration on Thursday approved BioMarin Pharmaceutical's gene therapy for severe hemophilia A, the company said, giving patients with the inherited bleeding disorder an alternative to regular injections of missing blood proteins. Analysis: Macro hedge funds down at half-year point, pin recovery hopes on central banks 2023-06-30T04:00:00Z The Food and Drug Administration approved Roctavian for adult patients with severe cases of hemophilia A, the inherited blood-clotting disorder that can lead to bleeding after minor injuries or scrapes. Gene therapy for severe hemophilia is approved by FDA 2023-06-29T04:00:00Z The Food and Drug Administration has approved a gene therapy treatment for adults with hemophilia B that costs $3.5 million per dose, making it the world’s most expensive medicine. FDA approves gene therapy for hemophilia B, now world’s costliest medicine 2022-11-24T05:00:00Z Earlier this year, European regulators approved a similar gene therapy for hemophilia A. That drug, from drugmaker BioMarin, is still under review at the FDA. $3.5M gene therapy for hemophilia gets FDA approval 2022-11-22T05:00:00Z The drug developer gave investors a potentially discouraging update on the development of a hemophilia drug. Bristol-Myers, Gilead rise, SVB Financial, BioMarin fall 2022-09-12T04:00:00Z If a mutation takes place in germ cells, the mutation will be passed on to the next generation, as in the case of hemophilia and xeroderma pigmentosa. Biology for AP Courses 2022-06-09T00:00:00Z A similar gene therapy approved last year for hemophilia B was priced at $3.5 million, making it the most expensive one-time treatment of its kind. Gene therapy for severe hemophilia is approved by FDA 2023-06-29T04:00:00Z Biopharmaceutical firm CSL Behring manufactures the gene therapy treatment Hemgenix, which allows patients with hemophilia to produce a protein that makes their blood clot in open wounds and halt prolonged bleeding. FDA approves gene therapy for hemophilia B, now world’s costliest medicine 2022-11-24T05:00:00Z The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder which primarily affects men. $3.5M gene therapy for hemophilia gets FDA approval 2022-11-22T05:00:00Z Mark Fox, who contracted Hepatitis C after being treated with a contaminated blood product for hemophilia as a child, said he welcomed the announcement as an acknowledgement of wrongdoing by the authorities. Victims of UK tainted blood scandal to get government payout 2022-08-17T04:00:00Z But how do genes located on a chromosome ultimately produce a polypeptide that can result in a physical phenotype such as hair or eye color—or a disease like cystic fibrosis or hemophilia? Biology for AP Courses 2022-06-09T00:00:00Z Scientists could cure diseases such as cystic fibrosis or hemophilia. Miller & Levine Biology 2018-01-01T00:00:00Z Some experts see the price as high but acknowledge that existing treatments for hemophilia are also expensive and say the fear of bleeding complications will drive patient demand. FDA approves gene therapy for hemophilia B, now world’s costliest medicine 2022-11-24T05:00:00Z Hemgenix is the first such treatment for hemophilia and several other drugmakers are working on gene therapies for the more common form of the disorder, hemophilia A.. $3.5M gene therapy for hemophilia gets FDA approval 2022-11-22T05:00:00Z This is the case in the genetic disorder hemophilia, which is actually a group of related disorders, the most common of which is hemophilia A, accounting for approximately 80 percent of cases. Anatomy and Physiology 2013-06-19T00:00:00Z The most common form of hemophilia affects 1 out of every 5,000 male births worldwide, but the condition is much rarer in females. Biology for AP Courses 2022-06-09T00:00:00Z “This is what happened with cancer and other rare diseases like hemophilia.” With a $2.1 Million Cure Their Only Hope, Parents Plead for Help Online 2022-04-06T04:00:00Z Advocates for hemophilia B patients were also pleased with the federal approval of the new treatment. FDA approves gene therapy for hemophilia B, now world’s costliest medicine 2022-11-24T05:00:00Z He also held lucrative leadership roles at pharmaceutical and biotech companies developing drugs for patients with hemophilia and impaired muscle function. F.D.A. Nominee Faces Steep Climb to Senate Confirmation 2022-02-03T05:00:00Z Patients with hemophilia bleed from even minor internal and external wounds, and leak blood into joint spaces after exercise and into urine and stool. Anatomy and Physiology 2013-06-19T00:00:00Z Plasma donations are typically used to develop medicine that helps people who suffer from rare and chronic complex conditions like PI or hemophilia. How you can help change lives and become a plasma hero | Provided by BioLife Plasma Services 2022-02-02T05:00:00Z The drug developer reported encouraging results from a study on a potential hemophilia treatment. Take-Two, Lululemon fall; Jazz, BioMarin rise 2022-01-10T05:00:00Z The company is working on therapies for hemophilia, narcolepsy and other conditions. Senators Question Califf, the F.D.A. Nominee, on Opioid Crisis and Pandemic Response 2021-12-14T05:00:00Z Donated plasma is most commonly used to treat patients with clotting factor disorders such as hemophilia and von Willebrand disease. Blood plasma isn’t effective therapy for covid, but it’s vital for other illnesses 2021-12-10T05:00:00Z Inadequate clotting can result from too few platelets, or inadequate production of clotting factors, for instance, in the genetic disorder hemophilia. Anatomy and Physiology 2013-06-19T00:00:00Z 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 The Advance Study “is an important piece of research that hopefully will give us comfort in advancing” further, said Skinner, who lives with severe hemophilia and became HIV-positive decades ago through a blood product. Giving blood has been off limits for many gay men. A new study could help change that 2021-07-06T04:00:00Z BioMarin Pharmaceutical Inc. in California is taking a similar approach with a different virus called AAV for hemophilia, a blood clotting disorder, and several other diseases. Newer methods may boost gene therapy’s use for more diseases 2021-06-01T04:00:00Z People with hemophilia, who also have low or absent levels of clotting proteins, face similar challenges. Blood plasma isn’t effective therapy for covid, but it’s vital for other illnesses 2021-12-10T05:00:00Z 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 The findings “support the conclusion that an AAV integration event was not likely responsible” for the liver tumor, says hemophilia gene therapy researcher Denise Sabatino of the Children’s Hospital of Philadelphia. Experimental gene therapy for hemophilia probably did not cause patient’s liver tumor 2021-03-29T04:00:00Z Nienhuis helped create a pediatric brain tumor program and a project to develop gene transfer for hemophilia treatment, the hospital said. Former St. Jude Children’s Research Hospital director dies 2021-02-09T05:00:00Z This year, the coronavirus pandemic disrupted that opportunity for these campers and their families, already all too familiar with battling diseases — from cancer and sickle cell anemia to hemophilia. Camp provides 'different kind of healing' to kids with serious illnesses 2020-12-23T05:00:00Z A treatment for hemophilia B that restores a missing protein called factor IX, developed by the company BioMarin, is close to winning U.S. regulatory approval. Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment 2020-12-22T05:00:00Z The Whites filed suit against one of the manufacturers of the blood products Ryan received three times each week for his hemophilia. Editorial Roundup: Indiana 2020-12-01T05:00:00Z The Food and Drug Administration wants more information on the drug developer’s potential hemophilia treatment before considering its approval. Target, Momenta Pharmaceuticals rise; BioMarin, TJX fall 2020-08-19T04:00:00Z It was named in honor of a brave boy from Indiana who contracted HIV from a treatment for his hemophilia and became a powerful public face for those suffering from the disease. I witnessed the horror of HIV 30 years ago. Here's how we can conquer a pandemic | Cleve Jones 2020-08-18T04:00:00Z Nelson, however, was classified 4-F because of a mild form of hemophilia, so he was unable to serve in uniform. Upcoming COVID charity events extend golf's long history of staging 'Challenge' matches - Golf Digest 2020-05-11T04:00:00Z UniQure’s hemophilia B treatment also appears to be working, with 52 of 54 patients no longer needing injections of factor IX after 6 months in its latest study. Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment 2020-12-22T05:00:00Z Ryan suffered from hemophilia, an inherited deficiency in the clotting of blood. Editorial Roundup: Indiana 2020-12-01T05:00:00Z Two years later, the Centers for Disease Control and Prevention announced that most cases were among gay men, intravenous drug users, Haitians and people with hemophilia. Will the coronavirus outbreak lead to new L.A. crime fiction? The jury is out 2020-03-30T04:00:00Z Food and Drug Administration last year for spinal muscular atrophy, a fatal childhood neurological disease, and a treatment for the blood-clotting disorder hemophilia B that’s expected to receive FDA approval this year. Virus used in gene therapies may pose cancer risk, dog study hints 2020-01-06T05:00:00Z While contending with the effects of hemophilia, Mr. Massie’s eldest son grew up to be an Episcopal priest, author, activist and Democratic politician. Robert K. Massie, award-winning historian of Czarist Russia, dies at 90 2019-12-03T05:00:00Z For years, he had been anxious to write about hemophilia. Robert Massie, who popularized Russian history, dead at 90 2019-12-03T05:00:00Z They follow Biomarin Pharmaceutical Inc, developer of a gene therapy for hemophilia, which constructed one of the industry’s largest manufacturing facilities in 2017. Pfizer, Novartis lead pharma spending spree on gene therapy production 2019-11-27T05:00:00Z A large number of patients who needed regular blood transfusions, many with hemophilia, contracted HIV, and many died. First New HIV Strain in 19 Years Identified 2019-11-06T05:00:00Z In the hemophilia treatment, AAV infects liver cells and turns the organ into a factory for making the clotting protein that patients depend on. Virus used in gene therapies may pose cancer risk, dog study hints 2020-01-06T05:00:00Z One of its projects is a gene therapy treatment for hemophilia. FTC staff recommends approval of Roche deal for Spark: report 2019-10-24T04:00:00Z Massie thought the film superficial, but took advantage of the publicity to raise money for hemophilia treatment. Robert Massie, who popularized Russian history, dead at 90 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 Drugs for hemophilia and other rare diseases are similarly pricey. The $6 Million Drug Claim 2019-08-25T04:00:00Z “All that effort we made to get here from Honduras and now we’re going back,” said Angel Estrada, who had hoped to get care in the United States for his 9-year-old son, who has hemophilia. Thousands of Central American migrants take free rides home... 2019-08-21T04:00:00Z A blood test confirmed Jack’s diagnosis of hemophilia A, his father, who declined to say where Jack was treated, said. UK boy with severe, unexplained bruising diagnosed with rare blood disorder after parents accused of abuse 2019-07-26T04:00:00Z Roche wants to buy Spark to, among other things, get ahold of U.S. firm’s experimental gene therapy for hemophilia A as well as its platform to develop other treatments for genetic diseases. Roche's $4.3 billion Spark bid delayed again amid U.S., UK scrutiny 2019-06-10T04:00:00Z SMA affects only a few thousand patients in the U.S., but gene therapies for hemophilia and other genetic diseases are on the horizon. Is $2.1 million Too Much for a Drug? For Affected Parents, There Is No Debate 2019-06-04T04:00:00Z 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 It's rare; roughly 400 babies are born with hemophilia A each year in the U.S. UK boy with severe, unexplained bruising diagnosed with rare blood disorder after parents accused of abuse 2019-07-26T04:00:00Z Before the A.C.A., people with conditions like cancer or hemophilia that were very expensive to treat often faced enormous out-of-pocket costs once their medical bills reached these caps. What Happens if Obamacare Is Struck Down? 2019-03-26T04:00:00Z The gene that directs production of CCR5 may be modified with newer gene therapy techniques, similar to treatments developed for hemophilia and sickle-cell disease. An H.I.V. Cure: Answers to 4 Key Questions 2019-03-05T05:00:00Z The New England Hemophilia Association says hemophilia and other inheritable bleeding disorders impact over 3 million Americans. Ceremony to bring awareness to bleeding disorders 2019-03-02T05:00:00Z Roche already sells Hemlibra against hemophilia A that helps stop bleeding in patients with the life-threatening genetic disorder that prevents their blood from clotting. Roche bets $4.3 billion on Spark in gene therapy move 2019-02-25T05:00:00Z “You do eventually adapt to life with hemophilia and, although it takes time, life becomes pretty normal.” UK boy with severe, unexplained bruising diagnosed with rare blood disorder after parents accused of abuse 2019-07-26T04:00:00Z Spark is a gene therapy company with a pipeline of potential products targeting blindness, hemophilia and neurodegenerative diseases. Roche nearing $5 billion deal to acquire Spark Therapeutics: WSJ 2019-02-23T05:00:00Z Bayer said it was pleased with the verdict, “which confirms the strength of Bayer’s innovation in hemophilia treatment.” U.S. jury orders Takeda to pay Bayer $155 million over hemophilia drug 2019-02-05T05:00:00Z In the past few years, gene-replacement techniques have advanced to the point where they can treat congenital blood disorders, such as hemophilia and thalassemia. The History of Blood 2019-01-07T05:00:00Z Sales of new multiple sclerosis, hemophilia and cancer drugs have been propelling Roche’s pharmaceuticals business in recent quarters. Roche Executive Daniel O’Day to Be Named Gilead CEO 2018-12-10T05:00:00Z His father was a bully and a tyrant, and the boy himself suffered from hemophilia, which kept him periodically housebound as a child and filled him with terror at the thought of bleeding to death. Review: The rise of Fox News is told through the man who made it happen in 'Divide and Conquer: The Roger Ailes Story' 2018-12-05T05:00:00Z Well beyond a feel-good philanthropic effort, BioMarin’s business strategy here is to build a long-term relationship with hemophilia patients who it wants to one day use its gene therapy. BioMarin sets stage early for hemophilia cure off-Broadway 2018-11-21T05:00:00Z In September, Switzerland-based Octapharma sponsored a free sea kayaking expedition for 10 hemophilia patients on Washington State’s Puget Sound, offering a chance to see pods of orca killer whales. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z Shortly after I completed my hematology training, in the early eighties, I encountered many people with hemophilia who were dying from blood transfusions they’d received. The History of Blood 2019-01-07T05:00:00Z Treatments for other diseases, including other vision disorders and hemophilia, are now in development. High Hopes for a Gene Therapy Come With Fears Over Cost 2018-09-24T04:00:00Z Likewise, cellular implants could transform treatment of other disorders, including cancer, heart failure, hemophilia, glaucoma and Parkinson’s disease. Implantable Drug-Making Cells 2018-09-19T04:00:00Z Some develop an immune response, like Porus, and their costs rise to more than $1 million per year, making hemophilia patients among some of the most expensive for the U.S. healthcare system. BioMarin sets stage early for hemophilia cure off-Broadway 2018-11-21T05:00:00Z Now, there are some two dozen approved products, with more in the pipeline as hemophilia’s potentially rich returns lure companies to pursue new approaches. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z Then the brothers joined Spark’s gene therapy trial for hemophilia B. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z That day, while biotechnology stocks overall fell, shares of hemophilia gene therapy manufacturers rose. FDA repays industry by rushing risky drugs to market 2018-07-02T04:00:00Z He then starred at B.U. under Parker, but during his junior season he was diagnosed with a form of hemophilia known as Christmas disease. David Quinn Comes to the Rangers With a Gift for Developing Young Players 2018-05-24T04:00:00Z Gottlieb said that hemophilia is the first disease the FDA will target with its new policy. FDA Plans to Speed Path to Approval for Some Gene Therapies 2018-05-23T04:00:00Z In 2016, the U.S. government alone spent some $2 billion on hemophilia A therapies, data from its Medicaid and Medicare insurance programs show. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z In May, he announced plans to approve gene therapies for hemophilia based on whether they increased the level of clotting proteins, without waiting for evidence of reduced bleeding. FDA repays industry by rushing risky drugs to market 2018-07-02T04:00:00Z He then played at Boston University, but as a junior he suffered bleeding in a leg and learned that he had Christmas disease, a form of hemophilia. Rangers on Verge of a Coaching Deal With Boston University’s David Quinn 2018-05-19T04:00:00Z An FDA spokesperson later said hemophilia was chosen because “it’s an area of a lot of development activity.” FDA Plans to Speed Path to Approval for Some Gene Therapies 2018-05-23T04:00:00Z Traditionally a cancer drugs maker, Roche is turning to rare diseases like hemophilia to replace revenue from its older medicines whose expiring patents have exposed the company to unprecedented generic competition. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z No one is saying yet that hemophilia will be cured. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z They were supposed to be a temporary solution, said Dana Kuhn, a hemophilia and HIV patient who founded PSI. Mother, wife, million-dollar patient 2018-04-25T04:00:00Z Some analysts and investors criticized the Baxalta deal because of new competition in the hemophilia market and the looming threat of a new kind of drug known as gene therapy. Fast-Growing Shire PLC Had Pharmaceutical Rivals in a Takeover Frenzy 2018-04-19T04:00:00Z Private Mulligan worked in a textile mill as a teenager in Greenville, S.C., before the war, and lied about his health to get into the Marines, hiding his hemophilia. Lost in Battle, Found by Amateur Sleuths: An ‘Unknown’ Marine 2018-04-17T04:00:00Z Michelle Rice, the NHF’s head of external affairs and the mother of a hemophilia patient, said convincing everyone in the hemophilia community to quickly switch therapies will be difficult, despite benefits promised by new drugs. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z There are 20,000 hemophilia patients in the United States who lack one of two proteins needed for blood to clot. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Medications for hemophilia are crucial to patients — overwhelmingly male — with the rare genetic condition that prevents clotting and puts them at great risk of bleeding to death, even from a minor injury. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z Last week, Sanofi agreed to buy U.S. hemophilia specialist Bioverativ for $11.6 billion, its biggest deal for seven years and a major play to strengthen its presence in treatments for rare diseases. France's Sanofi buys Ablynx for $4.8 billion in biotech M&A boom 2018-01-29T05:00:00Z Increasing competition in both cancer and hemophilia, coupled with those high price tags, means there is a real risk that both acquisitions will fail to generate good returns. High Prices Won’t Deter Biotech Deals 2018-01-22T05:00:00Z Many hemophilia patients died after acquiring HIV and hepatitis through tainted blood products in the 1980s. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z Even with regular injections, people with hemophilia risk uncontrolled bleeding into a muscle or joint, or even the brain. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Contributing to the costs is the fact that there is no cure for hemophilia and no cheaper substitute for blood factor. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z Last week, Sanofi agreed to buy U.S. hemophilia specialist Bioverativ for $11.6 billion, its biggest deal for seven years and a major play to strengthen its presence in treatments for rare diseases. France's Sanofi buys Ablynx for $4.8 billion in biotech M&A boom 2018-01-29T05:00:00Z Earlier this month, Sanofi said it obtained the right to develop and sell a new hemophilia drug in a restructuring of its partnership with Alnylam Pharmaceuticals. Sanofi set to buy Bioverativ for more than $11.5 billion: WSJ 2018-01-21T05:00:00Z A 2016 paper she co-authored described hemophilia’s corporate-patient links and how companies use gifts, financial opportunities and one-on-one marketing to advance their commercial interests. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z Even a routine flight is risky, said Mark Skinner, a 57-year-old attorney in Washington with hemophilia who is a past president of the World Federation of Hemophilia. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z And the Medicaid program's three most expensive drugs per prescription are for hemophilia, according to an analysis by the Kaiser Family Foundation. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z Now 50 years old, Martin was born with hemophilia, and he bleeds almost uncontrollably from a cut. Why Our Own Immune Systems Attack Our Best Drugs--And How to Stop It 2018-01-10T05:00:00Z Earlier this month, Sanofi said it obtained the right to develop and sell a new hemophilia drug in a restructuring of its partnership with Alnylam Pharmaceuticals. Sanofi set to buy Bioverativ for more than $11.5 billion: WSJ 2018-01-21T05:00:00Z As part of Roche’s initial marketing campaign for Hemlibra, it has produced videos of hemophilia patients participating in daily activities, including riding bikes, in spite of their serious illness. With Hemlibra, Roche seeks to break into tight hemophilia circle 2018-10-03T04:00:00Z People with hemophilia often are taught as children to avoid most sports and to find professions that will not require much physical activity. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Generally speaking, the price of hemophilia drugs rise as rival drugs hit the market. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z More than 560 clinical trials are under way testing gene- or cell-based therapies for a range of conditions including hemophilia and sickle cell disease, according to the American Society of Gene & Cell Therapy. FDA Blesses Blindness Treatment That Could Cost $1 Million 2017-12-19T05:00:00Z In a study published earlier this year, his team, along with scientists at Arcturus Therapeutics, treated hemophilia in mice using mRNA that encodes a clotting protein. Can a multibillion-dollar biotech prove its RNA drugs are safe for a rare disease? 2017-12-19T05:00:00Z Spark plunged and competitor BioMarin climbed after both reported data on experimental treatments for hemophilia A. Apple and Chesapeake Energy rise; Spark Therapeutics sinks 2017-12-11T05:00:00Z The clotting proteins used in the 1980s were contaminated with H.I.V. and hepatitis C. Nearly everyone with hemophilia got infected. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Caitlin Carroll, director of public affairs for PhRMA, the pharmaceutical industry lobbying group, said high development costs and a complicated and lengthy manufacturing process play a role in how hemophilia drugs are priced. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z The hemophilia gene therapy is one of several being tested for conditions including sickle cell anemia and Huntington's disease. Gene therapy makes a big advance treating hemophilia B blood disorder 2017-12-06T05:00:00Z Other companies are working on hemophilia treatments; Sangamo Therapeutics is testing traditional gene therapy and gene editing approaches. Gene therapy shows promise against blood-clotting disease 2017-12-06T05:00:00Z Many hemophilia patients rely on opioid painkillers because nearly every other pain reliever increases the risk of internal bleeding. APNewsbreak: Startup could bring back Vioxx for hemophilia 2017-11-21T05:00:00Z “It’s a really optimistic time,” said Dr. Lindsey A. George, a hematologist at the Children’s Hospital of Philadelphia and a principal investigator for Spark Therapeutics, one of several companies developing gene therapies for hemophilia. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z State health officials say the costs of hemophilia are hard to anticipate and control, even with rebates. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z “People were planning their lives around hemophilia, and now they are doing activities that they weren't before,” George said. Gene therapy makes a big advance treating hemophilia B blood disorder 2017-12-06T05:00:00Z Another gene therapy, from BioMarin Pharmaceutical for a different form of hemophilia, also showed promise in a different study. Gene therapy shows promise against blood-clotting disease 2017-12-06T05:00:00Z Amish men and women who inherit two copies of the PAI mutation, one from each parent, for example, produce no PAI-1 at all and develop a bleeding disorder similar to hemophilia. Amish Mutation Protects Against Diabetes and May Extend Life 2017-11-15T05:00:00Z Although the symptoms are the same with both forms of the disease, hemophilia A is by far the most common. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z In 2016, California's Medicaid program paid at least $205 million for medications used to treat hemophilia, according to a Kaiser Health News analysis of federal Medicaid data. Case of the $21-million Medicaid patient shows how drug prices soar for rare diseases 2018-03-05T05:00:00Z He said the data suggests that the treatment ultimately might provide an “ideal cure” for hemophilia B. Gene therapy makes a big advance treating hemophilia B blood disorder 2017-12-06T05:00:00Z Hundreds of gene-therapy clinical trials are underway throughout the world, including for hemophilia and Huntington’s disease, according to the Alliance for Regenerative Medicine, an industry group. FDA advisers urge approval for first gene therapy for inherited disease 2017-10-12T04:00:00Z Her son, Charles, 13, has spent much of life in and out of hospitals and doctors’ offices for grueling hemophilia treatments. ‘Progressive Liberal’ inside wrestling ring riles them up 2017-08-12T04:00:00Z Spark, with gene therapy for hemophilia B, and BioMarin, another biotech company, with a similar treatment for hemophilia A, are starting large, final-phase clinical trials. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z His company’s goal, he said, is to offer its still-in-the-works hemophilia therapy at a lower price than other treatments. If he wanted to, Trump could take steps to lower soaring drug prices 2017-08-01T04:00:00Z "Shire's goal with this action is to ensure the hemophilia community receives sufficient, accurate information," the British company said. Roche, Shire court fight underscores high stakes in hemophilia 2017-07-10T04:00:00Z Five years later it is being used to cure mice with HIV and hemophilia. The Gene Editors Are Only Getting Started 2017-07-07T04:00:00Z He was 77 and suffered from hemophilia, but no immediate cause was announced. Times Square, Roger Ailes, Saudi Arabia: Your Thursday Evening Briefing 2017-05-18T04:00:00Z Patients in Spark’s hemophilia B trial only reached on average 35 percent of normal blood levels of factor IX. But those levels have remained steady for the two years they have been followed. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z He had hemophilia yet his father sometimes beat him with a belt, according to Sherman’s biography. Roger Ailes, ousted founder of Fox News Channel, dies at 77 2017-05-18T04:00:00Z The group says an estimated 64,000 people in Massachusetts - and more than 3 million Americans overall - suffer from hemophilia and related disorders that prevent proper blood clotting in the body. Advocates, lawmakers seek awareness for hemophilia patients 2017-03-05T05:00:00Z In a statement, the hemophilia foundation said it never allows its corporate sponsors to influence its decision-making, and that it also does not endorse specific products or favor certain companies. More Than 80 Percent of Patient Groups Accept Drug Industry Funds, Study Shows 2017-03-01T05:00:00Z As for gene editing in patients’ cells that aren’t inherited, clinical trials are already underway for HIV, hemophilia, and leukemia. U.S. panel gives yellow light to human embryo editing 2017-02-14T05:00:00Z At first, hemophilia seemed ideal for gene therapy. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Desperate to find a cure for their ailing son’s hemophilia, one night they called upon Rasputin. 5 Myths and Truths About Rasputin 2016-12-29T05:00:00Z That forced the company to lower its earnings forecasts and subsequently announce a reorganization and spin-off of its hemophilia business. Investors see Biogen CEO choice as friendly to potential takeover 2016-12-20T05:00:00Z 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 It’s 1976, and up until this point, a diagnosis of hemophilia was basically a deferred death sentence, the inability to produce blood clots making any bleed — especially an internal one — a perilous prospect. ‘Roz and Ray’: A dad and a doctor locked in battle for boys’ survival 2016-10-20T04:00:00Z The genes for hemophilia A and B were isolated in the early 1980s. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z You've probably heard of hemophilia – with its history among European royalty – but maybe not about von Willebrand disease, the much more common bleeding disorder affecting up to 1 percent of the U.S. population. Von Willebrand Disease: the Under-the-Radar Bleeding Disorder 2016-10-07T04:00:00Z 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 Given the continuing contribution from the hemophilia business for the rest of the year, and following second-quarter results that easily exceeded projections, Biogen provided a full-year earnings forecast that topped analyst expectations. Biogen Says CEO George Scangos to Step Down 2016-07-21T04:00:00Z In May the company said it plans to spin off its hemophilia drug business into a separate publicly traded company, but it now expects to complete that move early next year. Biogen rises after 2nd-quarter results surpass forecasts 2016-07-21T04:00:00Z They treated ten patients with hemophilia B and managed to increase their blood levels of factor IX to between 2 percent to 6 percent of normal. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z There are college scholarships for people with the disease at multiple companies and paid internships, such as "Bayer Leadership U," which promises to "to help prepare future leaders in the hemophilia community." How drug companies use gifts and internships to buddy up to their most valuable patients 2016-06-14T04:00:00Z Gene therapy is showing promise for several rare diseases, including hemophilia B, in which people lack a livermade protein called factor IX that helps blood clot. How a liver-damaging drug may give a boost to gene therapy 2016-06-08T04:00:00Z As part of its move to narrow its focus, the company said in May that it would spin off its hemophilia drug business. Biogen Says CEO George Scangos to Step Down 2016-07-21T04:00:00Z The combined company, which will be run from Shire’s Dublin headquarters, aims to be a world leader in treating hemophilia and other rare diseases, and will benefit from Ireland’s lower corporate tax rate. Eight automakers recall over 12 million vehicles for defective air bag inflators 2016-05-27T04:00:00Z Investigators realized that they could put the mutated gene into a virus and use it to insert the mutated gene into the cells of patients with hemophilia B. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z But it also details other things drug companies offer to develop relationships with patients, such as grants to support swimming lessons or music classes for families with hemophilia. How drug companies use gifts and internships to buddy up to their most valuable patients 2016-06-14T04:00:00Z So far, it hasn’t given hemophilia B patients enough factor IX to completely avoid bleeding episodes—they need about 12% of normal levels, but get half or less with the therapy. How a liver-damaging drug may give a boost to gene therapy 2016-06-08T04:00:00Z Experts said both should stir discussion among doctors, patients and parents who deal with hemophilia. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z Her only son’s hemophilia, inherited through her grandmother Queen Victoria, left the empress ripe for the ministrations of a stunning succession of holy fools, ending with Rasputin. The Romanovs: fascinating, odd and odious 2016-05-13T04:00:00Z They cannot carry a large gene, and the gene for factor VIII, needed to treat hemophilia A, is enormous. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z "Patients with hemophilia face significant clinical, social, and economic challenges that impede access to care. Sometimes those needs aren’t met through private or public insurance," Inchausti said in an email. How drug companies use gifts and internships to buddy up to their most valuable patients 2016-06-14T04:00:00Z In January, Baidu faced scrutiny for allowing an unlicensed private hospital to host an online forum on hemophilia. Chinese search engine Baidu under investigation for ad placements after student's death 2016-05-05T04:00:00Z In the United States, about 20,000 people -- mostly boys and men -- are living with hemophilia, according to the U.S. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z In January, Baidu faced online fury after it sold the management rights to a bulletin-board platform for patients with hemophilia to a company that Internet users said populated the site with questionable information. China Launches Baidu Probe After the Death of a Student 2016-05-02T04:00:00Z Nathan Wilkes, 42, who lives in the Denver suburbs, said years of trying to get care for his son, who has hemophilia, had worn his family thin and made him an advocate for universal coverage. Colorado considers universal health-care proposal 2016-04-28T04:00:00Z Notably, these are around Shire’s big bet on hemophilia, an area marked by increasing competition and potential innovative new treatments. How Will Shire Heal After Biotech Bruising? 2016-04-14T04:00:00Z In February, U.S. regulators approved its Kovaltry, a drug that reduces bleeding episodes in hemophilia patients. Retooled Bayer expands US business 2016-04-01T04:00:00Z "That worked well until the 1980s and the HIV epidemic," said Dr. Christopher Walsh, director of the hemophilia program at Mount Sinai Hospital, in New York City. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z Monahan and his colleagues also compared men with severe hemophilia to those with mild hemophilia. Review Finds Mixed Success With Hemophilia Treatment 2016-03-16T04:00:00Z Most of the spending was driven by new specialty drugs, which treat diseases that affect relatively few people, such as hemophilia or end-stage cancer. Voter anger over surging prescription drug costs has generated a campaign issue 2016-03-11T05:00:00Z Food and Drug Administration to replace a deficient blood clotting factor among people with hemophilia B. Idelvion Approved for Hemophilia B 2016-03-07T05:00:00Z All of my friends, and some of my family too, wanted to help raise awareness for hemophilia.” New Jersey boy becomes Marvel’s new superhero 2016-01-23T05:00:00Z The new trial tested the drug in just 18 patients with severe hemophilia. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z The researchers said they were surprised to find that frequent bleeding was common even among the youngest men with severe hemophilia. Review Finds Mixed Success With Hemophilia Treatment 2016-03-16T04:00:00Z One of those children was a young Iowan with hemophilia identified by the court as “A.K.” Recent editorials published in Iowa newspapers 2016-01-18T05:00:00Z People with the rare genetic disorder hemophilia B are prone to recurring episodes of serious bleeding, primarily into the joints. Idelvion Approved for Hemophilia B 2016-03-07T05:00:00Z More and more, manufacturers of hemophilia drugs and the specialty pharmacies that dispense the medicines are hiring patients and their relatives to gain an inside track and access in selling their products. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z Both studies tackle a key challenge: Up to one-third of children with severe hemophilia develop antibodies against the standard therapy. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z The companies said their combined hemophilia franchise will provide a foundation for a broader hematology and oncology business. Business digest: Irish drugmaker closer to Baxalta deal; fantasy sports may keep operating 2016-01-11T05:00:00Z Iowa’s largest health insurer searched for any way to justify not reimbursing an Iowa City pharmacist for drugs he dispensed to hemophilia patients. Recent editorials published in Iowa newspapers 2016-01-18T05:00:00Z There also will remain lingering doubts about the durability of Baxalta’s business in hemophilia treatments, which represents the bulk of its earnings, and where new competitors and technologies loom. Shire’s Baxalta Deal: Confidence Knocked 2016-01-11T05:00:00Z In the Alabama case, the owners of the specialty pharmacy MedfusionRx were convicted after prosecutors charged that they hired a man with hemophilia to get him and his relatives with the disease as customers. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z Between the late 1970s and mid-1980s, half of Americans with hemophilia became infected with HIV through contaminated blood products, according to the National Hemophilia Foundation. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z The first gene-repair clinical trials, for hemophilia and cancer, are already getting underway. A look ahead at 2016: What research trends will be hot—and what will not? 2015-12-30T05:00:00Z Additionally, the agency said people with hemophilia and related blood clotting disorders will continue to be banned from donating blood due to potential harm they could suffer from large needles. FDA overturns 30-year ban on blood donations by gay men 2015-12-22T05:00:00Z The agency said people with hemophilia and related blood-clotting disorders will continue to be banned from donating blood due to potential harm they could suffer from large needles. FDA drops lifetime ban on blood donations from gay and bisexual men 2015-12-21T05:00:00Z Pharmacies that specialize in hemophilia drugs, like BioRx and Factor Support Network, are being snapped up by larger pharmacies. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z The ultimate hope, he noted, is to use gene therapy to potentially cure hemophilia. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z He questioned the logic of Shire exposing itself unnecessarily to the risky hemophilia market. Standoff Over Shire’s Pursuit of Baxalta Nears an End 2015-12-16T05:00:00Z The therapies in the pipeline are mostly for rare genetic diseases: sickle cell, hemophilia or immune deficiency. Gene therapies offer dramatic promise but shocking costs 2015-11-11T05:00:00Z But, financial appeal aside, questions linger over the strategic merits of Baxalta’s hemophilia business, as well as the risks of digging in for a protracted hostile battle. Shire’s Baxalta Vision May Be Fading 2015-10-19T04:00:00Z The phenomenon of patients working for the industry is not unique to hemophilia, but is much more common for that disease, according to various patient advocates. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z An international team of researchers randomly assigned 264 young children newly diagnosed with severe hemophilia to start replacement therapy with either blood-derived or recombinant factor VIII. 2 New Findings Offer Hope for Those With Severe Hemophilia 2016-05-25T04:00:00Z Baxalta specializes in treatments for the bleeding disease hemophilia and immune deficiencies. Shire Weighing Options to Sweeten All-Stock Offer for Baxalta 2015-09-13T04:00:00Z The attorney general’s office said that Trinity agreed to repay $2.5 million last month in an investigation related to hemophilia drugs. NY says home care company will pay $22.4M in inquiry 2015-07-10T04:00:00Z They also believe the company’s drug pipeline in oncology and biosimilars is strong enough of offset and revenue headwinds if Roche, Alnylam Pharmaceuticals or a gene therapy specialist successfully enter the hemophilia market. Baxalta And Ebay's PayPal Are The Best Spinoffs To Target This July 2015-07-02T04:00:00Z Also convicted was the mother of a young man with hemophilia, who was paid 45 percent of the profit MedfusionRx made from each customer she recruited. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z Rasputin was a Russian mystic who first gained the confidence of Tsarina Alexandra and then the Tsar himself, thanks to his ability to ease the suffering of their son Alexei, who suffered from hemophilia. 7 Times Game of Thrones Drew on History This Season 2015-06-15T04:00:00Z Mike, a native of Bolingbrook, was diagnosed shortly after birth with afibrinogenemia, a bleeding disorder similar to hemophilia. EXCHANGE: Student fights back from stroke suffered at age 18 2015-06-08T04:00:00Z “As much as we worry about premature or ethical use, using these technologies to get rid of genetic disease would be amazing: Tay-Sachs, cystic fibrosis, sickle cell disease, hemophilia,” Caplan said. Scientists are growing anxious about genome-editing tools 2015-05-18T04:00:00Z At the time, Dr. Johnson and other researchers were experimenting with gene therapy for disorders like hemophilia. Protection Without a Vaccine 2015-03-09T04:00:00Z The owners of a specialty pharmacy in Alabama were convicted of inflating bills to Medicaid by paying huge commissions to some members of the hemophilia community to recruit patients. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z The company did notch three approvals, including two promising hemophilia drugs. Grading Pharma In 2014: 17 Drug Companies Ranked 2015-01-04T05:00:00Z Although Spark’s gene therapy platform initially will focus on treatment of hemophilia B, there exists the potential for developing similar therapies for the treatment of cancer. Pfizer Moving Into Gene Therapy Is A Welcome Move 2014-12-10T05:00:00Z And while she never deals in controlled substances like painkillers and opiates, she does collect medicines to treat diabetes, scabies, heart disease and even hemophilia. Recycling unused meds for overseas a gray area 2014-12-02T05:00:00Z In both trials, the drug caused minimal side effects, and it was efficacious at treating hemophilia in one trial as well. Surviving the Rare Disease Roller Coaster 2014-11-20T05:00:00Z In the past, drug companies and pharmacies routinely took patients to dinner and provided presents like sleeping bags to children at hemophilia summer camps. Hemophilia Patient or Drug Seller? Dual Role Creates Ethical Quandary 2016-01-13T05:00:00Z With all this interbreeding, difficulties inevitably arose, such as the hemophilia that was passed on to several of Victoria’s children, especially the unhappy Leopold. Queen for many a day Spark is currently investigating the efficacy of gene therapy for hemophilia B, and its program will enter early phase trials next year. Pfizer Moving Into Gene Therapy Is A Welcome Move 2014-12-10T05:00:00Z About half the cases of acquired hemophilia are related to other medical conditions, such as pregnancy, cancer or use of certain medications. New Treatment Approved for Rare Form of Hemophilia 2014-10-24T04:00:00Z The company develops, manufactures and markets products that focus on hemophilia, immune disorders, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. Is Capitalism's Immune System Strong Enough To Fight Ebola? 2014-10-21T04:00:00Z Gene therapy is now being developed to treat a range of disorders, from hemophilia to Parkinson’s to a deadly neurological illness called Sanfilippo syndrome. Biotechnology to the Rescue 2014-09-02T04:00:00Z He was born with a severe form of hemophilia. My Mother’s Miracle-Water Cure 2014-07-11T04:00:00Z Each year, about 120,000 infants are born with problems including heart defects, blood disorders such as hemophilia or sickle cell disease, physical abnormalities or cancer, according to the U.S. What to Do When Your Baby Is Born With a Health Problem 2014-05-27T04:00:00Z Unlike the more common form of hemophilia that's inherited and affects males, acquired hemophilia affects both males and females. New Treatment Approved for Rare Form of Hemophilia 2014-10-24T04:00:00Z Biogen's announcement of its donation coincides with its launch of new hemophilia drugs, the Globe reported. Health Highlights: May 13, 2014 2014-05-13T04:00:00Z The companies last month received U.S. approval for Alprolix, which treats the more rare hemophilia B. Biogen, Sobi pledge hemophilia drug donation in developing world 2014-05-12T06:05:41Z Dan, who suffers from hemophilia, a disorder in which the blood does not clot properly, had been infected with HIV through a transfusion when he was 12. As mixed-status HIV couples weigh risks, more choose to conceive the old-fashioned way 2014-04-24T22:13:09Z Patients with more severe hemophilia require larger and more frequent doses of blood clotting factors. Exclusive: Biogen prices hemophilia drug on par with older therapies 2014-04-18T17:04:29Z Its portfolio includes recombinant and plasma-based proteins to treat hemophilia and other bleeding disorders, and plasma-based therapies to treat immune deficiencies, burns and shock, and other blood-related conditions. DealBook: Baxter to Split Into 2 Health Care Companies 2014-03-27T12:47:10Z A U.S. biotechnology company says it will donate hundreds of millions of dollars worth of hemophilia drugs to developing nations. Health Highlights: May 13, 2014 2014-05-13T04:00:00Z The success of the humanitarian effort will hinge on Food and Drug Administration approval of Eloctate for hemophilia A, which would account for at least 85 percent of the donated product. Biogen, Sobi pledge hemophilia drug donation in developing world 2014-05-12T06:05:41Z Hemophilia A is caused by a lack of clotting factor VIII, while hemophilia B is caused by a lack of clotting factor IX. New hemophilia drugs aim to simplify treatment 2014-03-07T12:06:46Z Denmark's Novo Nordisk is also developing a long-acting treatment for hemophilia B, and expects to file next year for regulatory approval. Exclusive: Biogen prices hemophilia drug on par with older therapies 2014-04-18T17:04:29Z Though my father was a pediatrician and my mother a nurse, they didn’t even consider hemophilia. A Conversation With Holbrook Kohrt: A Doctor’s Intimate View of Hemophilia 2013-12-23T22:51:43Z People with hemophilia lack proteins required to control bleeding. Health Highlights: May 13, 2014 2014-05-13T04:00:00Z Food and Drug Administration will extend by three months its deadline for reviewing the company's experimental long-acting medicine for hemophilia. Biogen says FDA extending review of hemophilia drug 2013-12-02T14:01:06Z Baxter expects to file by the end of this year for U.S. approval of its long-lasting hemophilia A treatment. New hemophilia drugs aim to simplify treatment 2014-03-07T12:06:46Z Worldwide, about one in 5,000 men are born with hemophilia A and one in 25,000 men are born with hemophilia B each year. Exclusive: Biogen prices hemophilia drug on par with older therapies 2014-04-18T17:04:29Z Once that was known, my parents became centered on taking care of a child with severe hemophilia. A Conversation With Holbrook Kohrt: A Doctor’s Intimate View of Hemophilia 2013-12-23T22:51:43Z A person with severe hemophilia may use up to 5,000 units of clotting factor a week. Health Highlights: May 13, 2014 2014-05-13T04:00:00Z 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 Worldwide, about one in 5,000 men is born with hemophilia A and one in 25,000 men is born with hemophilia B each year. New hemophilia drugs aim to simplify treatment 2014-03-07T12:06:46Z Alnylam is testing its technology in conditions as varied as liver cancer, respiratory tract infections, hemophilia and high cholesterol. Alnylam bets on 'gene silencing' to woo biotech investors 2013-07-12T14:47:25Z Many people there did not understand about hemophilia. A Conversation With Holbrook Kohrt: A Doctor’s Intimate View of Hemophilia 2013-12-23T22:51:43Z Rixubis, which is administered twice weekly, aims to control and prevent bleeding and better manage hemophilia B patients in the hospital for surgery. U.S. FDA approves new Baxter drug for hemophilia B 2013-06-27T21:28:22Z The study included 36 patients with hemophilia A or B and inhibitors over a 12-month period. Baxter posts positive results from late-stage hemophilia study 2013-01-08T16:31:38Z For hemophilia A, the advantages of the new products are less dramatic and uptake will be slower, Dr Young said. New hemophilia drugs aim to simplify treatment 2014-03-07T12:06:46Z Sometimes, they can be quite damaging: cancer, hemophilia and cystic fibrosis can all be caused by genetic mutation. How To Get Superpowers 2012-12-13T16:11:46Z Breakthroughs in biochemistry promised the same for hemophilia. A Conversation With Holbrook Kohrt: A Doctor’s Intimate View of Hemophilia 2013-12-23T22:51:43Z The most common form is called hemophilia A and occurs in more than 400,000 people worldwide, according to the National Hemophilia Foundation. U.S. FDA approves new Baxter drug for hemophilia B 2013-06-27T21:28:22Z "Sport and physical activity is beneficial for children with hemophilia but the benefits must be balanced against the increased risk of bleeding episodes, particularly bleeding into muscles and joints," Broderick said. Sports only small risk in rare bleeding disorder 2012-10-09T20:12:17Z Meanwhile, biotechnology companies like Sangamo BioSciences and BioMarin Pharmaceutical are working on methods to replace defective hemophilia genes, allowing patients to begin producing their own clotting factor. New hemophilia drugs aim to simplify treatment 2014-03-07T12:06:46Z The company is also testing a drug for the more common hemophilia A, and expects to present results from that trial by the end of the year. Biogen's hemophilia drug meets late-stage trial goal 2012-09-26T20:40:09Z It expects to present results from the hemophilia A trial by the end of the year. Biogen's hemophilia drug meets late-stage trial goal 2012-09-26T14:24:15Z Hemophilia B is the second most common type of hemophilia, which affects 3,300 people in the United States and some 25,000 worldwide, according to the National Hemophilia Foundation. U.S. FDA approves new Baxter drug for hemophilia B 2013-06-27T21:28:22Z Despite recent successes – LCA2, hemophilia, adrenoleukodystrophy — the pharmaceutical industry has been hesitant to fund gene therapy because it has lacked an approval. Rare Diseases: 5 Recent Reasons to Cheer 2012-07-29T22:15:05.603Z The annual market for hemophilia treatments is estimated at $8 billion worldwide. Ipsen suffers setbacks on hemophilia drug trials 2012-07-10T10:10:15Z The top five diagnoses: cancer, heart disease, degenerative bone diseases, organ failure requiring a transplant and hemophilia. Fate of 'uninsurables' hinges on Supreme Court 2012-05-17T21:24:40Z An even more striking manifestation of a physical anomaly, as a family trait, is the condition known as hemophilia. Essays In Pastoral Medicine 2012-03-05T03:00:09.993Z The company reported positive results in animal testing for its hemophilia drug, according to a statement issued yesterday. Hacker Attacks a Boon for Nice as ADRs Rally: Israel Overnight 2012-02-15T01:17:39Z When you belong to a disease group that is as small as hemophilia, it’s best that you provide some example that you exist, and that you’re living a full life. Patient Voices: Changed, but Not Defined, by Hemophilia 2012-01-30T20:19:27Z The disease primarily affects men, and patients with hemophilia take injections of a clotting factor to prevent or reduce bleeding episodes. Well Blog: Patient Voices: Hemophilia 2012-01-25T20:23:32Z There are several types of hemophilia, including hemophilia A and B. About 80 percent of hemophilia cases are hemophilia A, caused by defects in the blood-clotting agent Factor VIII. Gene therapy shows promise in treating hemophilia 2011-12-11T01:33:03Z The two main forms of the disease are hemophilia A, caused by a lack of clotting factor VIII, and hemophilia B, caused by a lack of clotting factor IX. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z About 80 percent of hemophilia cases are of the type known as hemophilia A, which is caused by defects in a different blood-clotting agent, Factor VIII. Hemophilia B Gene Therapy Breakthrough 2011-12-10T21:55:07Z At nytimes.com/health, five people living with hemophilia talk about how the condition affects their lives. Patient Voices: Changed, but Not Defined, by Hemophilia 2012-01-30T20:19:27Z People with severe hemophilia usually must get injections of a clotting-factor drug two to three times a week. Gene Therapy Allows Hemophiliacs to Skip Weekly Shots in Study 2011-12-10T17:35:08Z The study was preliminary and involved only six patients, and other promising early attempts to use gene therapy against hemophilia ultimately failed. Gene therapy boosted blood-clotting for hemophiliacs in small study called ?terrific advance? 2011-12-10T16:20:14Z Researchers have so far treated six men with severe hemophilia B who were producing clotting factor IX at less than 1 percent of normal levels. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z The general goal of current hemophilia treatment is to achieve factor levels greater than 1 percent of normal. Gene therapy proves effective for hemophilia B 2011-12-10T16:12:22Z Its medications are used to treat people with hemophilia, kidney disease, immune system problems, infectious diseases, serious burns and other conditions. Lawyer for Mexico arm of US drugmaker Baxter recorded allegedly offering payment in lawsuit 2011-08-17T07:25:52Z The test may be particularly valuable for families that harbor sex-linked genetic disorders like hemophilia, they add. Mother's blood test reveals baby's sex 2011-08-09T20:26:17Z Dr. High said it was too soon to try the technique in people, given that an adequate treatment for hemophilia already exists. Hemophilia Is Target of Therapy on Genome 2011-06-27T05:58:17Z Since the gene is carried on the X chromosome, hemophilia is almost exclusively a disease of men. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z Hemophilia B is much less common than hemophilia A, a deficiency of clotting factor VIII. Gene therapy proves effective for hemophilia B 2011-12-10T16:12:22Z If Donald suspected that knowledge would translate into fetal selection and subtraction, he probably envisioned women attempting to avoid debilitating sex-linked diseases like hemophilia. How Ultrasound Changed the Human Sex Ratio 2011-06-11T13:45:00.243Z Food and Drug Administration, the researchers found there is little evidence that the drug, called NovoSeven and marketed by the company Novo Nordisk, actually helps prevent death in people who have conditions other than hemophilia. Hemophilia drug often used in other patients: study 2011-04-18T21:18:06Z New analyses suggest that rFVIIa's widespread use outside hemophilia has no merit. Hospitals Engaging in Risky Use of Clotting Drug 2011-04-18T22:00:00Z Hemophilia B is much less common than hemophilia A. About one in five hemophilia patients has hemophilia B, according to the National Institutes of Health. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z Worldwide, about 1 in 5,000 men are born with hemophilia A and 1 in 25,000 men are born with hemophilia B each year. Gene therapy proves effective for hemophilia B 2011-12-10T16:12:22Z Tests for other conditions, including cystic fibrosis, Tay-Sachs, hemophilia and sickle cell disease, may be four to five years away. Too Much Information? Noninvasive Genetic Tests for the Unborn 2011-04-13T12:45:02.793Z In people with hemophilia, blood takes a long time to clot. Hemophilia drug often used in other patients: study 2011-04-18T21:18:06Z An expensive clotting drug approved to treat only hemophilia has become extremely popular in hospitals to stem bleeding during heart surgery, brain hemorrhages, trauma, liver transplants, and prostate removal. Hospitals Engaging in Risky Use of Clotting Drug 2011-04-18T22:00:00Z Worldwide, about one in 5,000 men is born with hemophilia A and 1 in 25,000 men is born with hemophilia B each year. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z Patients with hemophilia lack, or are deficient in, certain proteins called clotting factors that help stop bleeding. Biogen to build blood disorder drug franchise 2011-03-04T16:42:26Z I know how you look down on our small size, our allergies and hemophilia and all the other weaknesses that have been bred back and preserved by the race. Sense of Obligation 2011-02-09T03:00:52.337Z Just 20,000 people in the United States have hemophilia, according to an estimate by the Centers for Disease Control and Prevention. Hemophilia drug often used in other patients: study 2011-04-18T21:18:06Z He was given a diagnosis of hemophilia, treated and placed in the hospital's NICU, where he received treatment to stop the bleeding and remained under observation for a day. Health insurance plans sometimes don't cover care needed by newborn babies 2011-01-04T04:10:03Z After every procedure, young patients being treated for cancer, sickle cell, hemophilia and other ailments select the appropriate bead to mark another milestone reached. John Kelly: Special necklaces mark progress of cancer patients 2009-12-02T05:00:00Z The idea of building a blood disorder franchise around Biogen's hemophilia drugs is relatively new. Biogen to build blood disorder drug franchise 2011-03-04T16:42:26Z Gerus-Darbison and others in the hemophilia community stand against America's gay-rights community, the Red Cross, the American Medical Association and others. HIV's tragic legacy haunts blood-donor discussion 2010-04-10T00:33:00Z The finding helped doctors understand why women suffer less often from genetic diseases that tend to affect men, like hemophilia. Dr. Barton Childs, Who Studied Inherited Diseases, Is Dead at 93 2010-03-09T06:00:00Z Consider von Willebrand disease, an inherited disorder that — like the better-known hemophilia — is caused by a deficiency in one of the essential factors that govern the blood’s ability to clot. 18 and Under: When a Scratch or a Nosebleed Turns Into a Flood 2010-03-08T23:05:00Z I worked directly with them and the entire hemophilia community. New at the Top: Wendy K.D. Selig, Melanoma Research Alliance 2010-03-01T05:00:00Z The two main forms of the disease are hemophilia A, caused by a lack of clotting factor VIII, and the less-common hemophilia B, caused by a lack of clotting factor IX. Biogen to build blood disorder drug franchise 2011-03-04T16:42:26Z Set against the feelings of the hemophilia community is a rising chorus of calls for change. HIV's tragic legacy haunts blood-donor discussion 2010-04-10T00:33:00Z "You mean something like the reverse of hemophilia?" Earth Alert! Abnormally heavy menstrual flow, or menorrhagia, is another common problem for girls and women with von Willebrand disease — which, unlike hemophilia, is not a sex-linked trait. 18 and Under: When a Scratch or a Nosebleed Turns Into a Flood 2010-03-08T23:05:00Z Such is the case with insanity, with epilepsy, with hemophilia, or "bleeders," and with certain rare and curious disturbances of the nervous system, such as the hereditary ataxias and "tics" of various sorts. Preventable Diseases Kolster has investigated hemophilia in women, and reports a case of bleeding in the daughter of a hemophilic woman. Anomalies and Curiosities of Medicine On the other hand, they said, anemias, tuberculosis, hemophilias, scrofulas occurred more among the lymphatic type. The Glands Regulating Personality Fortunately the functions of menstruation and parturition are not seriously interfered with in hemophilia. Anomalies and Curiosities of Medicine Queen Victoria, herself unaffected, was a carrier — thus that famous pedigree by which royal children with hemophilia married into so many ruling families of Europe. 18 and Under: When a Scratch or a Nosebleed Turns Into a Flood 2010-03-08T23:05:00Z |
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