News

The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…

The global pharmaceutical company Mylan has voluntarily issued a nationwide recall of four lots of tranexamic acid injection — a generic form of Cyklokapron — and of amiodarone HCl injection, according to a company announcement published by the U.S. Food and Drug Administration (FDA). The…

In rare instances, people with hemophilia can grow bones outside their skeletal tissue — with bone growth found in muscles and soft tissues — which significantly limit their range of motion, according to a recent case report. Surgery can improve the functional outcomes of patients with this abnormal bone…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Genentech has launched a program called HemeWork to support the professional development and career goals of people living with hemophilia. HemeWork was developed with input from the Hemophilia Federation of America (HFA) and bleeding disorder advocates across the U.S. to help members of the bleeding…

The National Hemophilia Foundation (NHF) has received a $500,000 grant from the Centers for Disease Control and Prevention (CDC) to support education and outreach efforts in bleeding disorders. The five-year funding will help the NHF conduct programs for patients and healthcare providers related to inhibitor awareness, treatment for…

Tiny particles containing RNA led to successful delivery and production of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — in a mouse model of the disease, according to a new study. The study, “Functionalized lipid-like…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…

The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others in the hemophilia community by surprise. That decision, announced in a complete response letter of…