The Muscular Dystrophy Association (MDA) has opened applications for its 2026 college scholarship program, which provides financial support for students living with muscular dystrophy or a related neuromuscular disease. With the program now in its third year, the scholarships are available to students who are pursuing or planning…
Note: This story was updated Oct. 20, 2025, to correct the reported increase in dystrophin levels observed at higher doses of brogidirsen in the Phase 1/2 study. Brogidirsen (NS-089/NCNP-02), NS Pharma’s investigational therapy for people with Duchenne muscular dystrophy (DMD) who are amenable to exon 44 skipping, has…
An investigational gene therapy called GNT0004Â has demonstrated sustained stabilization of motor function at up to two years of follow-up in two boys with Duchenne muscular dystrophy (DMD). The boys received what was determined to be the effective dose of GNT0004, which was developed by Genethon, during the initial…
Parent Project Muscular Dystrophy (PPMD), a nonprofit dedicated to ending Duchenne muscular dystrophy (DMD), has published an updated edition of the PJ Nicholoff Steroid Protocol, marking the 10th anniversary of its first release. Called PJ’s Protocol, the guidance is designed to help healthcare providers manage corticosteroid use…
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