| 单词 | hemophilia B |
| 例句 | 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 |
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