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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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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…
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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,…
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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…
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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…
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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…
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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…
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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…
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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…
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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.
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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,…
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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…
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