Columnist Jennifer Lynne interviews Debbie Murray, a New Yorker whose hemophilia B diagnosis didn't occur until she was almost 51.

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?

Hemophilia is caused by deficiency of clotting factors, a certain type of protein in the body. Major symptoms include unexplained bleeding, blood in urine or stool. The condition is diagnosed by ...

Srivastava and colleagues said their study validated a new approach to gene therapy in severe hemophilia A -- through transplantation, after myeloablative conditioning, of autologous HSCs ...

Concizumab-mtci (Alhemo) is approved for hemophilia A or B with inhibitors, offering a subcutaneous alternative to intravenous treatments. The phase 3 explorer7 trial demonstrated an 86% reduction ...

Severe hemophilia A is caused by a genetic absence ... potentially countering the confounding of the previous study. With such a small sample size and short follow-up time, more research will ...

Elsewhere on the hemophilia front, Novo is looking to challenge Roche’s bleeding disorder stalwart Hemlibra, which generated nearly $4.8 billion in sales last year.

This production improves thrombin generation and clot formation in patients with hemophilia A or B with inhibitors. The approval is supported by data from the multicenter, open-label phase 3 ...