The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne muscular dystrophy (DMD). “The initiation of our pivotal trial, which is the first Phase 3 DMD gene therapy program to begin enrolling eligible participants, is an important milestone for the…
When I’m deep in thought, reflecting on my life, I shudder to think who and where I’d be without the support of my family and friends. Whether I need a shoulder to lean on or simply a sounding board for my own jumbled thoughts, I can trust that…
The investigational gene therapy SRP-9001 safely leads to the production of a functional form of dystrophin in boys with Duchenne muscular dystrophy (DMD) — and to improvements in their motor abilities, according to top-line results of a Phase 2 trial. However, following almost one year of the Sarepta…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
Since my internship at The Washington Post, I’ve done freelance work at outlets ranging from The Forward, a Jewish-focused website, to Dot Esports, and of course, Bionews, the publisher of Muscular Dystrophy News. In the past year and a half, I’ve realized that working for…
AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy and safety of the investigational medication AMO-02Â (tideglusib) in children and adolescents with congenital myotonic dystrophy type 1Â (CDM1), also known as Steinert disease. The double-blind trial (NCT03692312) is…
Throughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, clinical trial findings, and other relevant events related to muscular dystrophy. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a…
Edgewise Therapeutics has received $95 million in funding to advance the clinical development of its experimental therapy EDG-5506, designed to improve physical functioning in people with Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD).
Christmas is upon us, and for many, it might not even feel like the holiday season at all. We’ve been forced to stay away from family, our finances are tight, and uncertainty lingers about how 2021 will unfold. For me, being cooped up…
The French National Agency for Medicines and Health Products Safety has cleared Généthon to begin a clinical trial testing the safety and efficacy of GNT0004, a potential gene therapy for Duchenne muscular dystrophy (DMD), in France. “From translational research to clinical application, Généthon has supported this candidate drug,…
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