Gene Therapy for Treatment of Duchenne Muscular Dystrophy

EP: 1.Clinical Presentation and Diagnosis of Duchenne Muscular Dystrophy

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

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

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EP: 4.Gene Therapy for Treatment of Duchenne Muscular Dystrophy

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

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

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.