Sarepta plans to ask the FDA for full approval of exon-skipping Duchenne muscular dystrophy treatments Amondys 45 and Vyondys 53.
Early trial data suggest RNA therapies SRP-1001 and SRP-1003 reach muscle and show biomarker activity in muscular dystrophy.
Columnist Shalom Lim is using his lived experience with Duchenne muscular dystrophy to consult with organizations about ...
Dyne Therapeutics' Z-basivarsen led to gains in motor function and cognition for myotonic dystrophy type 1, and is entering a ...
New presentations at MDA 2026 expand the understanding of how Duvyzat may help people with Duchenne muscular dystrophy.
Dyne Therapeutics is advancing z-rostudirsen toward approval for DMD after data showed increased dystrophin and early ...
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of ...
The evidence for Elevidys is growing, and Sarepta is working to make the DMD treatment widely available, the company's ...
The MDA's annual conference highlighted recent breakthroughs and the outlook for future care in the neuromuscular disease community.
Capricor has reapplied for FDA approval of deramiocel, its therapy for DMD-related heart disease, and expects a decision by ...
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