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…
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The first participant was dosed with the investigational therapy marstacimab in a Phase 3 clinical trial — still recruiting eligible patients across the globe — in adolescents and adults with severe hemophilia A or B. Pfizer‘s marstacimab (PF‐06741086) is a human antibody that blocks a…
The healthcare insurance company BCS Financial has partnered with the National Hemophilia Foundation (NHF) to improve care quality and cost for people with hemophilia. BCS Financial provides services primarily for Blue Cross and Blue…
AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased factor IX (FIX) activity and controlled bleeding — while markedly reducing the need for other treatments — in the Phase 3 HOPE-B clinical trial, top-line data show. These findings will be presented at…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
A Phase 4 trial investigating Ixinity as a prophylactic treatment for children under age 12 with hemophilia B has reached the halfway mark for its enrollment target, Medexus Pharmaceuticals announced. The international study (NCT03855280), taking place at sites in Brazil, Colombia, Moldova, South Africa, and Ukraine, continues recruiting eligible…
The Dietary Approach to Stop Hypertension (DASH) diet yields beneficial health effects, reducing blood pressure, sugar, and fat, and increasing high-density lipoprotein (HDL) levels or so-called “good” cholesterol, in adolescents with hemophilia, an Iranian…
Due to the identification of new adverse events, Sanofi Genzyme has placed a dosing hold on its full clinical development program for fitusiran, an investigational treatment for hemophilia. The decision was announced in a joint statement from the World Federation of Hemophilia, the European…
Manual therapy, in combination with passive stretching, can reduce the frequency of joint bleeds and improve joint health in people with hemophilia, a small study shows. The study, “The effectiveness of manual therapy in addition to passive stretching exercises in the treatment of patients with haemophilic…
Using a combination of genetic and functional approaches, scientists have identified new mutations in parts of the F8 gene that do not code for factor VIII (FVIII) in a small group of French and Canadian patients with hemophilia A. These results were described in the study, “The…
