Hope for boys with Duchenne Muscular Dystrophy (DMD) has arrived with SRP-9001, the first FDA-approved gene therapy for this challenging condition. But what exactly is SRP-9001 (Delandistrogene moxeparvovec), and how does it work?
Discover how this innovative treatment works at the genetic level, potentially offering a life-altering solution for those affected by this debilitating condition. Take a look at the findings from the latest clinical trial, and the promising results that have sparked excitement within the medical community.

References:
1. Manini A, Abati E, Nuredini A, Corti S, Comi GP. Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence. Front Neurol. 2022;12:814174. Published 2022 Jan 5. doi:10.3389/fneur.2021.814174
2. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed February 13, 2024. [ Ссылка ]
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4. Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics. October 30, 2023. Accessed February 13, 2024. [ Ссылка ]
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#Elevidys #SRP9001 #DuchenneMuscularDystrophy #MedicalBreakthroughs #HopeForDMD #genetherapy #healthtech #biotechnology #DelandistrogeneMoxeparvovec

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