People with hemophilia A who start on preventive treatment in the first years of life generally report better health-related quality of life and joint health, a study reports. “Our results indicate that delayed start of prophylaxis in an older cohort with severe [hemophilia A] can still achieve excellent bleeding…

Almost two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce their quality of life, even when they’re on factor replacement therapy to reduce the frequency of bleeding episodes, a real-world study shows. “Overall, the data indicate that joint problems still…

Excessive bleeding after oral surgery led to two people in China, both with normal pre-operative blood clotting tests, being diagnosed with hemophilia A, according to a recent case report. Mild hemophilia can go undetected in standard laboratory tests, and oral surgeons need to be prepared for such cases, the research…

SCT800, a replacement therapy containing a man-made form of clotting factor VIII (FVIII), appears to be safe and effective at treating and preventing bleeds in adolescents and adults with severe hemophilia A, a study has found. The study, “Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted…

A Sangamo Therapeutics‘ gene therapy program to potentially treat hemophilia A, called SB-525, has been cleared by the U.S. Food and Drug Administration (FDA) for clinical testing in adult patients. The company announced that the FDA approved its Investigational New Drug (IND) application for SB-525, a necessary first step…

The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for eligible adults with severe hemophilia A.  Roctavian now becomes the first gene therapy to win FDA approval for the treatment of hemophilia A in the U.S. “Today’s approval of Roctavian builds…

The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of increasing levels…

Adults with mild and moderate hemophilia A have a substantial number of bleeds per year as well as joint problems and surgeries, revealing an unmet medical need in these patients, according to an interim analysis of the CHESS II study. The findings were shared at…

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…

A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model of hemophilia A, a study reported. The study, “Treatment of…