News

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…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient perceptions. These patients reported that they choose their treatments based mainly on five criteria. Specifically, those are…

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…

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…