麻豆传媒

First once-weekly subcutaneous prophylactic option for hemophilia B

Fidanacogene elaparvovec reduces bleeding episodes by 71% after a single infusion

No factor VIII inhibitors developed among the 74 boys included in the study

Tens of thousands contracted HIV or hepatitis from transfusions during the 1970s and 1980s

One dose of fidanacogene elaparvovec proved noninferior to standard prophylactic factor IX

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Uptake of new options for hemophilia A and B has been a bit slow

Marstacimab reduced the annualized bleeding rate versus on-demand, prophylactic treatments

More than half of children in study had no treated bleeds at follow-up of more than 100 weeks

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"Strategy will probably be practice-changing," says expert

Phase III trial shows 86% reduction in treated bleeds in hemophilia A and B with inhibitors

First gene therapy approved for severe hemophilia A

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