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.
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Episodes in this series
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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Related to this article
In recognition of International Ataxia Awareness Day, observed annually on September 25, NeurologyLive® reviews 6 investigational agents in clinical development across ataxia-related disorders.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 25, 2026.
One-year KYSA-6 data showed KYV-101, a CD19 CAR T-cell therapy, produced durable, drug-free responses in generalized myasthenia gravis.
The FDA has granted Scholar Rock's apitegromab fast track designation for the treatment of patients with living facioscapulohumeral muscular dystrophy as the company's phase 2 FORGE study dosing begins.
Ulefnersen met its primary end point in the phase 3 FUSION trial, providing the first placebo-controlled evidence for a genetically targeted therapy in FUS-ALS.
The CHMP recommended approval of gefurulimab (Klygefa), a weekly, self-administered C5 inhibitor, for AChR-antibody-positive generalized myasthenia gravis in the EU.
Here's some of what is coming soon to NeurologyLive® this week.
The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.
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