News

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ Phase 1/2 study evaluating the investigational therapy SIG-001 in adults with severe or moderately severe hemophilia A. The move comes after a serious adverse event (SAE) occurred involving one of the three participants…

The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…

Preventive treatment with Hemlibra (emicizumab) sustainably improved physical health-related quality of life and led to fewer missed workdays among people with severe hemophilia A without inhibitors, an analysis of pooled data from two clinical trials found. According to researchers, these findings add to previous data showing that Hemlibra…

For boys and men with moderate to severe hemophilia, switching to extended half-life (EHL) products is linked to a reduced number of infusions and a sustained low rate of bleeds, according to a  real-life, multi-center study in Canada. Notably, a significant drop in the annualized bleeding rate (ABR)…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…

BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. That resubmission, to the European Medicines Agency (EMA), included one-year follow-up data from BioMarin on the therapy’s safety and effectiveness in treating the rare genetic…

Complication rates for elective surgeries in people with inherited bleeding disorders like hemophilia are low in Australia, reflecting good safety in their performance despite guidelines not always being followed, a study reported. Use of a specialty hemophilia treatment center (HTC) for surgery, and use of factor replacement therapy were among guidelines sometimes…

Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found. The study, “Patients’ and parents’…

After one year, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to safely and effectively increase factor IX (FIX) activity and prevent bleeds in men with moderate to severe hemophilia B, according to new data from the Phase 3 HOPE-B trial. The men participating in the study did…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…