Gene therapy for Duchenne muscular dystrophy
Yuko Shimizu‐Motohashi · 2025 · Brain and Development
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract
Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disorder caused by variants of the DMD that leads to progressive muscle degeneration. Recent advances in gene therapy have opened new therapeutic avenues, particularly through the use of adeno-associated virus (AAV)-mediated micro-dystrophin delivery. Delandistrogene moxeparvovec, the first FDA-approved gene therapy for DMD, has demonstrated transgene expression and potential functional improvement in early phase trials, although its long-term efficacy, durability, and safety remain unconfirmed. Immune-mediated toxicities including
Abstract by Yuko Shimizu‐Motohashi, Brain and Development (2025) — licensed CC BY 4.0.
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Metadata source: OpenAlex · DOI 10.1016/j.braindev.2025.104424