News

SerpinPC, an investigational treatment that Centessa Pharmaceuticals is developing for hemophilia, will advance to interventional Phase 2 clinical testing later this year. The studies will support Centessa’s registrational program for hemophilia B. Such a program involves clinical trials that ultimately will lead to the company seeking regulatory approval…

Despite preventive treatment, people with hemophilia A or B report lower quality of life, mainly due to poor joint health, a study in Sweden shows. No significant difference was found between these two patient groups, who report high frequency of pain, mobility problems, and mental health issues like anxiety…

Proteins from tardigrades — microscopic animals known for their ability to survive in extreme conditions — could be used to stabilize replacement therapies for hemophilia A, allowing treatments to be stored for longer periods of time without refrigeration, a proof-of-concept study shows. “Our work provides a proof of principle…

For children with severe hemophilia A who develop inhibitors — neutralizing antibodies that reduce the effectiveness of standard therapies — starting treatment to eliminate inhibitors earlier is associated with a higher chance of success, a recent study reports. The study, “Low-dose immune tolerance induction therapy in…

People with moderate-to-severe hemophilia and ankle joint disease reported significant impairments in life quality and foot and ankle function, according to a recent U.K. study. Chronic pain emerged as a significant predictor of these patient-reported outcomes, as did the presence of inhibitors — neutralizing antibodies that can lower the…

Hemgenix (etranacogene dezaparvovec), the first gene therapy approved for adults with hemophilia B, has been shown to work better than standard preventive replacement therapy to lower the frequency of bleeds — both spontaneous and joint bleeds — in this patient population. The therapy also was superior in…

MRI screening can identify subclinical joint bleeding in some patients with severe hemophilia A on preventive treatment, according to a study in the Netherlands. Subclinical bleeds occur without symptoms and usually cannot be detected by standard physical and ultrasound examinations. “These MRI findings support the hypothesis that subclinical…

The U.S. Food and Drug Administration (FDA) is extending its review of BioMarin Pharmaceutical’s request to approve the gene therapy Roctavian (valoctocogene roxaparvovec) for adults with severe hemophilia A. A decision had been expected by the end of this month, but that date has now been…

Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…

Conditions like hemophilia and von Willebrand disease are thought to affect more than 3 million U.S. residents. Across the country, patients, caregivers, advocates, and other supporters are coming together this month for Bleeding Disorders Awareness Month (BDAM) to participate in conversations and webinars, and share information about key community…