Opinion
Video
Gene Therapy for Treatment of Duchenne Muscular Dystrophy
Author(s):
Focusing on gene therapy and exon skipping medications, an expert on Duchenne muscular dystrophy discusses current and emerging treatment options.
Video content above is prompted by the following questions:
- Provide an overview of delandistrogene moxeparvovec, micro-dystrophin gene therapy, that received accelerated approval for Duchenne muscular dystrophy (DMD) in 2023.
- Discuss ongoing studies for fordadistrogene movaparvovec gene therapy and other exon skipping therapies.
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