Opinion|Videos|July 3, 2024

Gene Therapy for Treatment of Duchenne Muscular Dystrophy

Author(s)Emma Ciafaloni, MD

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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