A new natural history study that seeks to collect information about how Becker muscular dystrophy (BMD) progresses in the absence of treatment is now enrolling. The study is being led by the GRASP (General Resolution and Assessments Solving Phenotypes) consortium and Virginia Commonwealth University, its primary sponsor. Also collaborating…
Twins Oscar and Sebastian Spink, 11, may not be your typical “Avengers” superheroes, but they are certainly pursuing a gargantuan task that would have impressed even Tony Stark. The boys, diagnosed with facioscapulohumeral muscular dystrophy (FSHD) in 2019 and die-hard fans of the Marvel Cinematic Universe, are attempting to…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle strength and motor function in the first three boys enrolled in a Phase 1 clinical trial. Based on these positive results of the therapy’s low dose, with follow-up ranging from one…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
The first healthy volunteer has been dosed with an investigational exon-skipping therapy, called PGN-EDO51, for Duchenne muscular dystrophy (DMD) in a Phase 1 trial, the treatment’s developer, PepGen, has announced. The single-ascending dose trial is investigating the treatment’s safety and tolerability in about 40 healthy adult males in Canada.
Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this year, with a filing expected to be completed by the end of June. The submission of the companies’ rolling new drug application (NDA) to the U.S. Food and Drug Administration…
Thirteen new hospitals and health institutions have been added to the Muscular Dystrophy Association’s MDA Care Center Network, expanding the network’s reach across the United States. These additions, which comprise more than 150 care centers, include 10 new grant awards as well as three care affiliates that have been…
Electrical muscle stimulation that mimics resistance training exercises improves muscle structure, function, and survival rates in a Duchenne muscular dystrophy (DMD) zebrafish model, a new study reveals. “The longstanding consensus in the muscular dystrophy field is that minimizing resistance training preserves muscle strength and mass because it lowers the…
When it comes to making decisions about how care goals should change for adolescent and young adults with Duchenne muscular dystrophy (DMD) as the disease progresses, informal conversations among family members are more common than discussions with clinicians, according to a study based on interviews with DMD patients and…
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