A protein called GCN5 is vital for normal muscle health and helps to coordinate the expression of several important muscle proteins, including dystrophin, according to a study done in mice. The results suggest that targeting GCN5 may be a useful therapeutic avenue in diseases of the muscles, such as…
The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical trial evaluating DYNE-251, an experimental exon-skipping therapy for Duchenne muscular dystrophy (DMD). Dyne now hopes to have that Phase 1/2 trial underway by June, the company said in a …
Cognitive problems seen in some Duchenne muscular dystrophy (DMD) patients may be associated with a shift toward the amyloidogenic pathway in memory-specific brain regions, according to a study in mouse models of the disease. The amyloidogenic pathway is a signaling cascade that leads to the formation of beta-amyloid, the…
The U.S. Food and Drug Administration (FDA) has approved Regenxbio’s request to launch a Phase 1/2 clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne muscular dystrophy (DMD). The study, called AFFINITY DUCHENNE and anticipated to start in…
Children with Duchenne muscular dystrophy (DMD) who had been given a placebo in a clinical trial experienced a marked improvement in motor abilities after being treated with Sarepta Therapeutics‘ experimental gene therapy SRP-9001, new top-line data show. “We are delighted to report positive results for Part 2 of…
The experimental oral therapy EDG-5506 is generally safe and results in rapid and profound reductions in the levels of muscle damage biomarkers in men with Becker muscular dystrophy (BMD), top-line data from a Phase 1b clinical trial show. “We believe the magnitude of reduction in multiple key biomarkers of muscle…
Muscular Dystrophy News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to muscular dystrophy (MD) throughout 2021. We look forward to reporting more news to patients, family members, and caregivers dealing with MD during 2022. Here are the top 10 most-read articles of…
Genetic medicines company Gennao Bio will use a $1 million CureDuchenne Ventures investment to help expand its gene monoclonal antibody (GMAB) platform technology, which is now applied to rare muscle disorders, including Duchenne muscular dystrophy (DMD). The initial focus of the nonviral delivery system Gennao Bio is…
When his daughter, Meredith Huml, was diagnosed — finally — with facioscapulohumeral (FSHD) muscular dystrophy in 2004, Raymond Huml immediately jumped into action, researching everything he could about the disease and reaching out to the FSHD Society. “We went to the FSHD Society and looked up what they…
A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has only been identified as a young man who was not able to walk. In a statement, Pfizer expressed…
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