News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…

The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…

Mobility problems, bleeding, pain, and uncertainly in daily life are the biggest challenges people with hemophilia are facing, according to a survey from six central European countries. Additionally, while most respondents, including caregivers, felt well informed on hemophilia, they continue to seek further education on the condition and new…

The first participant has been dosed in a Phase 1/2 clinical trial evaluating marzeptacog alfa activated (MarzAA) to treat bleeding episodes in patients with factor VII (FVII) deficiency, Glanzmann thrombasthenia, and hemophilia A with inhibitors receiving treatment with…

Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals.  These are…

Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases. These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States:…

AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with pre-existing antibodies against the treatment’s viral carrier, according to new trial data. Data from the Phase 3 HOPE-B trial suggest that nearly all hemophilia B patients, regardless of pre-existing viral…

The first participant has been dosed in a Phase 3 clinical trial testing marzeptacog alfa activated (MarzAA), an experimental under-the-skin therapy for hemophilia A and B patients with inhibitors. The study, called Crimson-1 (NCT04489537) and sponsored by the therapy’s developer, Catalyst Biosciences, is currently recruiting patients at sites…