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Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond

BACKGROUND: Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder which leads to progressive muscle degeneration and weakness. Most patients die from cardiac or respiratory failure. Gene transfer therapy offers a promising approach to treating this disorder. OBJECTIVE: Given the genet...

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Detalles Bibliográficos
Autores principales: Elbashir, Haitham, Fathalla, Waseem, Mundada, Vivek, Iqbal, Mehtab, Al Tawari, Asmaa A, Chandratre, Saleel, Bastaki, Laila, Romany, Ingy, Ismayl, Omar, Abou Tayoun, Ahmad
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9697043/
https://www.ncbi.nlm.nih.gov/pubmed/36245386
http://dx.doi.org/10.3233/JND-221528