Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400…
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Also covering MDA Conference 2026:
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne…
Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to DMD’s natural course,…
The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (DMD), significantly slows the progression of arm and heart damage in boys…
Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the…
Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial. The…
SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels of microdystrophin — a version of the muscle-protecting protein that’s deficient in DMD — and preserve muscle health for…
PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in an early clinical trial, with biomarker data suggesting…
Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had stable motor function over several years of follow-up, new data showed. That stands in contrast to the typical…
At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.
Eating more protein was associated with better lower limb function and a higher quality of life among people with muscular dystrophy (MD), regardless of their ability to walk, according to a recent analysis. However,…
Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the latest advances in neuromuscular disease research…