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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.

Episodes in this series

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 1 Clinical Presentation and Diagnosis of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 2 Communication of Duchenne Muscular Dystrophy Diagnosis and Coordination of Care

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 3 Current and Emerging Treatment Options and Management of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 4 Gene Therapy for Treatment of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 5 Targeting Pathways Downstream of Dystrophin: Vamorolone and Givinostat in Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 6 Improvement in Management of Duchenne Muscular Dystrophy in Recent Years

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 7 Multidisciplinary Care in Duchenne Muscular Dystrophy

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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