Patients and experts in limb-girdle muscular dystrophy (LGMD), along with drug developers, community leaders, and regulatory agency personnel, came together for the LGMD Scientific Workshop to discuss how to tackle the unmeet therapeutic needs of people with these muscle-wasting diseases. The workshop, held in early February in Maryland,…
A cell-based study revealed the biological mechanism behind the increased tendency for people with myotonic dystrophy type 2 (DM2) to develop autoimmune diseases. Researchers found that the genetic defect that causes DM2, called a repeat expansion, indirectly triggered an abnormal, antiviral immune response in patient cells. “That was our…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC 1044, an experimental therapy for people with Duchenne muscular dystrophy (DMD) caused by mutations that are amenable to exon 44 skipping. The FDA gives the designation to treatments with the potential to improve care…
SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). Developed by Satellos Bioscience, the therapy is the lead treatment candidate for Duchenne muscular dystrophy (DMD), with the first clinical trial of SAT-3247 for DMD…
EDG-5506, an oral treatment being developed by Edgewise Therapeutics, has been granted fast track designation for Duchenne muscular dystrophy (DMD) by the U.S. Food and Drug Administration (FDA). The agency gives this designation to experimental therapies that have the potential to fill unmet medical needs, with the…
Cognitive function in boys with Becker muscular dystrophy (BMD) appear to be stable over time, although these children may struggle with working memory and executive function, a small study reports. Based on its findings, the researchers are calling for boys with BMD to have access to routine cognitive and…
With projects spanning gene therapy research, patient advocacy, and air travel safety, seven U.S. organizations will receive $140,000 in overall funding from the Muscular Dystrophy Associations (MDA) Advocacy Collaboration Grant Program. The program, which opened in 2022, seeks to support and enhance key public policy and advocacy initiatives…
It may take an average of 11 years before people with Duchenne muscular dystrophy (DMD) lose upper body function and the ability to breathe on their own, according to a study by researchers of the HERCULES project, which set out to construct a natural history model of the…
A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to renew the conditional marketing authorization for Translarna (ataluren), PTC Therapeutics’ medication for Duchenne muscular dystrophy (DMD). The recommendation by the Committee for Medicinal Products for Human Use (CHMP) comes after the company appealed…
SRP-5051 is able to increase the production of dystrophin protein in people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping — a trial finding its developers suggest will lead to a positive risk-benefit profile for the next-generation DMD treatment. The full results of the two-part…
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