Real-World Experience With Duchenne Muscular Dystrophy Gene Therapy - CGTLive®
Elevidys, an AAV-based gene therapy for Duchenne muscular dystrophy, received FDA approval in 2023 for ambulatory patients aged 4-5 with specific DMD gene mutations, and expanded approval in 2024 for broader patient groups. While not curative, it offers a functional gene version, but faces challenges like insurance coverage and long-term data needs. Experts highlight the importance of patient education and choice amidst expanding treatment options.
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The FDA's approval of Sarepta Therapeutics' gene therapy product Elevidys adds complexity to DMD treatment options. Dr. John Brandsema discusses the potential benefits and risks, emphasizing the need for more data, especially from older patients. Elevidys aims to restore a functional dystrophin protein, but its irreversible nature and potential safety events complicate decisions. The therapy's eligibility and durability are still under study, particularly in non-ambulatory populations.
Elevidys, an AAV-based gene therapy for Duchenne muscular dystrophy, received FDA approval in 2023 for ambulatory patients aged 4-5 with specific DMD gene mutations, and expanded approval in 2024 for broader patient groups. While not curative, it offers a functional gene version, but faces challenges like insurance coverage and long-term data needs. Experts highlight the importance of patient education and choice amidst expanding treatment options.
At the 2024 MDA Clinical and Scientific Conference, presentations highlighted new DMD treatments, including gene therapy advancements and real-world Elevidys use, with discussions on safety risks and potential solutions like new delivery vehicles to reduce adverse events.