Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference,…
Articles by Lindsey Shapiro, PhD
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after…
Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a…
Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive…
Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the…
SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels of microdystrophin — a…
At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central…
Eating more protein was associated with better lower limb function and a higher quality of life among people with…
Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted…
Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in…