单词 | hemophilia A |
例句 | 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 |
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