US EditionAAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy
- The American Academy of Neurology released an Evidence in Focus article on May 14, 2025, reviewing delandistrogene moxeparvovec for Duchenne muscular dystrophy treatment.
- Delandistrogene moxeparvovec, a gene therapy administered as a single infusion, received FDA approval in June 2024 to treat males aged four years and older diagnosed with Duchenne muscular dystrophy, a condition characterized by progressive muscle weakness.
- Clinical studies noted the therapy failed primary motor function outcomes but may slow muscle decline; side effects include inflammation, liver injury, low platelets, and potential death.
- The gene therapy requires a single administration priced at $3.2 million, excluding expenses related to hospitalization and ongoing monitoring, and experts highlight the importance of continued research to evaluate its long-term effectiveness and safety.
- The article aims to support neurologists and families in understanding delandistrogene moxeparvovec’s use while noting it does not cure Duchenne but may stabilize disease progression.
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AAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy
To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology.
·United States
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High hopes and high costs: Exploring advances in Duchenne muscular dystrophy gene therapy
To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article in Neurology.
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