Mim8, an investigational therapy to prevent bleeding episodes in people with hemophilia A, is safe and well-tolerated at multiple doses, according to data from the Phase 1/2 FRONTIER1 trial. The therapy also showed signs of efficacy, with most patients given higher doses experiencing no bleeds during treatment. “We are…
Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…
European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…
Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in which it did so varied, a recent study reported. Researchers noted that while findings support the use of this…
Symptoms of pain, anxiety, and depression generally increased with disease severity in people with hemophilia and were also associated with a lower quality of life, according to a recent survey involving patients, caregivers, and healthcare providers. While many surveyed individuals felt their pain was adequately addressed in the clinic, fewer…
CSL Behring will donate 500 million international units (IUs) of coagulation factor therapy to the World Federation of Hemophilia (WFH) in an effort to promote reliable access to treatment for bleeding disorders, including hemophilia, across more than 60 developing countries. The donation marks the fifth multiyear…
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…
Structural joint damage and significant pain are evident in the ankles of people with hemophilia, but the two were not found to be related to one another in a recent study. Researchers suspect that ankle pain may originate in the somatosensory nervous system, which relays nerve signals from the…
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