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. www.businesswi...
3. www.fda.gov/ne...
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. investorrelati...
5. www.musculardy...
Disclaimer: This KZitem video is not intended to provide diagnosis, treatment, or medical advice. The content provided in this video is for informational purposes only. Please consult with a physician or other healthcare professional regarding any medical or health-related diagnosis or treatment options. Information on this video should not be considered as a substitute for advice from a healthcare professional. The statements made about specific products are not to diagnose, treat, cure, or prevent disease.
#Elevidys #SRP9001 #DuchenneMuscularDystrophy #MedicalBreakthroughs #HopeForDMD #genetherapy #healthtech #biotechnology #DelandistrogeneMoxeparvovec
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