Jesy Nelson, type 1 and Spinal Muscular Atrophy
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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 the novel development of AAV gene therapy and its ...
CORAL GABLES, Fla. — There’s new hope for children born with a fatal form of muscular dystrophy. Nicklaus Children’s Hospital is one of the first in the nation and one of only three hospitals in Florida offering Elevidys, an FDA approved infusion ...
Neurological disorders affect the function of the nervous system. Common pediatric neurological disorders include ADHD, autism spectrum disorder, epilepsy, and cerebral palsy. Neurological disorders are conditions that affect the nervous system. Recent ...
Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and breaking trial findings.