Duchenne Muscular Dystrophy: Could Gene Therapy Be the Answer? Exploring SRP-9001

Описание: 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. https://www.businesswire.com/news/hom...
3. https://www.fda.gov/news-events/press...
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. https://investorrelations.sarepta.com...
5. https://www.musculardystrophyuk.org/n...

Disclaimer: This YouTube 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