Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

With ongoing breakthroughs in gene and RNA therapies, coupled with regulatory encouragement and AI-based innovation, the market for treating rare neurological diseases is on the cusp of a ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

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A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

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One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...

The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

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Biogen Inc. ( NASDAQ: BIIB) Stifel 2025 Virtual CNS Forum Conference March 18, 2025 10:15 AM ET Great. Good morning, everybody. Happy to be moderating my annual panel where Priya has to listen to me ...

Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy. The patient ...