Marta Figueiredo, PhD, managing science editor —

Mild-to-moderate hemophilia has a significant impact on the physical and psychosocial health, and life quality, of both men and women, according to a recent study. Findings also highlighted the unmet needs of these patient groups, supporting better guidelines and quality of care for people with mild-to-moderate disease, its researchers…

The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A. The designation is “a critical program to advance the efficient development and regulatory review of regenerative medicine products that have the…

The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

CSL Behring has issued a voluntary, pharmacy level recall covering one batch of Mononine, its long-standing treatment for preventing and controlling bleedings in people with hemophilia B that is soon to be discontinued. According to the company’s statement, the recall is a precautionary measure due to…

People with severe hemophilia B are likely to develop hemophilic arthropathy — a painful and degenerative joint disease caused by recurrent bleedings — despite the lower bleeding frequency relative to hemophilia A, a natural history study shows. The data also highlights that prophylactic (preventive) treatment guidelines are not being…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), due to a possibly related serious adverse event in one patient. The event concerns a preliminary diagnosis of hepatocellular carcinoma (HCC), a form…

When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, according to follow-up data from the Alta Phase 1/2 clinical trial. All five men given the highest dose…

AMT-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and the use of clotting factor IX (FIX) in men with moderate to severe hemophilia B, long-term data from a Phase 1/2 clinical trial show. The therapy also prevented…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…