News

The one-time gene therapy BBM-H901 (dalnacogene ponparvovec) has been officially approved in the Macao region of China for the treatment of adults with moderate to severe hemophilia B, thus expanding access to the medication beyond mainland China. Developed by Belief Biomed (BBM), BBM-H901 became China’s first and only…

A small survey study in Germany found that many people with severe hemophilia are not interested in receiving gene therapy, despite having “only general knowledge” of the treatment option — which, the researchers noted, could be “a potential cure with a single infusion.” According to the scientists, these…

In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…

A single infusion of the experimental gene therapy GS001 safely increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A  — for almost three years, effectively reducing bleeds and the need for replacement therapies at doses much lower than those used in currently approved…

Many boys and young men with hemophilia regularly participate in sports. However, doing so frequently — particularly three or more times a week — may raise the risk of sports-related bleeding, especially during early adolescence, a Japanese study reports. “Regardless of sports type, most bleeding episodes happened during junior…

A single infusion of Beqvez (fidanacogene elaparvovec-dzkt), a now-discontinued gene therapy for hemophilia B, led to sustained production of factor IX (FIX) — the clotting protein missing in people with the condition — for up to six years, reducing bleeding episodes and eliminating the need for routine preventive…

Children with hemophilia in areas with limited medical resources face a high risk of fatal brain bleeds, a danger that is dramatically worsened by delays in seeking emergency care. A new study from Thailand reveals that without access to modern preventive therapies, more than a third of pediatric patients…

An experimental therapy may help remove harmful antibodies that reduce the effectiveness of factor replacement therapies in hemophilia A, potentially offering a new way to overcome one of the most significant complications of standard care. The therapy uses chimeric autoantibody receptor T cells, or CAAR-T cells. These are…

In boys and men with severe hemophilia A, starting prophylaxis — preventive treatment given regularly to reduce bleeding — early and maintaining regular physical activity were each linked to a lower risk of hemophilic arthropathy, a progressive form of joint damage caused by repeated bleeding, according to a real-world…

A young woman who sought emergency care for severe pain and swelling of her left hand five weeks after giving birth was ultimately diagnosed with acquired hemophilia A — caught early enough that she did not require surgery. That’s according to a new report detailing the woman’s case, which,…