Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...

Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression of the high-activity factor IX ...

Pfizer said Wednesday that its gene therapy for hemophilia A successfully reduced patients’ bleeds for at least 15 months in a large Phase 3 trial. The data may be enough to muster approval, but they ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

Less than a year after approval, Pfizer said it plans to discontinue its hemophilia B gene therapy fidanacogene elaparvovec (Beqvez) across all global markets, citing weak demand. "Pfizer has made the ...

Earlier this year we reported on FDA's approval of valoctocogene roxaparvovec (Roctavian) for adults with severe hemophilia A. In this story, we provide an update on what has happened since. Two gene ...

However, investigators said there is some evidence suggesting attention-deficit/hyperactivity disorder may be more common in certain age groups of children with ...

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Hemophilia A is a rare, inherited condition that prevents your blood from clotting the way it should. People with hemophilia A do not make enough of a protein called factor VIII, which normally works ...

Blood clotting represents one of the body’s most elegant and complex protective mechanisms. This intricate process involves numerous proteins working in precise sequence – often described as a ...

Hemophilia may lead to internal bleeding, particularly in joints and the brain, which could cause pain, swelling, and neurological symptoms. Preventive treatment with clotting factor concentrates and ...