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Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their EXPLORE44® clinical trial, which is assessing the safety and efficacy of the investigational therapy delpacibart zotadirsen (formerly AOC 1044, abbreviated as del-zota) in people living with Duchenne who are amenable to exon 44 skipping. The Avidity team shared background information…

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The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the first three people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakening in the throat and eyes — in a clinical trial. That’s according to interim data shared at the Muscular Dystrophy Association‘s 2025…

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The neuromuscular junction (NMJ) disorders are a group of conditions that disrupt the communication between motor neurons and muscles, resulting in muscle weakness, fatigue, problems with movement and mobility, and, in severe cases, paralysis. Recognized NMJ disorders include the autoimmune disorders myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS), the genetic conditions known as congenital myasthenic…

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Researchers at AskBio are seeking adults with genetically confirmed limb-girdle muscular dystrophy (LGMD) type 2I/R9 to participate in a phase 1/2 clinical trial (LION-CS101) to evaluate the safety and tolerability of the investigational gene therapy AB-1003 to treat LGMD2I/R9. LGMD2I/R9 is a rare form of LGMD caused by changes in the FKRP gene and is associated…

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PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his role as Director of the FDA’s Center for Biologics Evaluation and Research (CBER). In his resignation letter, addressed to Acting FDA Commissioner Sarah Brenner, Dr. Marks indicated that his departure will be effective April 5, 2025. This leadership change is…

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Adding more movement or physical activity to each day is a common goal among people living with neuromuscular disease. However, muscle fatigue, weakness, and limited strength or range of motion can make increasing physical activity a challenge. The age-old conundrum of “if you don’t use it, you lose it” conflicts with the need for rest…

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Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung function for boys with Duchenne muscular dystrophy (DMD), new analyses indicate. The studies were presented in a series of posters at this year’s meeting of the Muscular Dystrophy Association (MDA). Duvyzat is an oral therapy developed…

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​Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of individuals with Duchenne who are amenable to exon 53 skipping. According to Wave, FORWARD-53 achieved all trial goals, demonstrating sustained exon skipping, muscle concentrations, and dystrophin restoration through…

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I was tempted to do a play on the show “America’s Got Talent” and title this column “FSHDers got talent,” but I knew the improper grammar wouldn’t get past my excellent editors. That being said, after participating in a recent FSHD Society wellness group session, I know that my fellow FSHDers — those of us…

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A team has discovered a possible therapeutic approach to repair injured muscles either from aging or degenerative muscle disorders.

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