Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the DMD gene and is characterized by progressive muscle degeneration that results in loss of function and early death due to respiratory and/or cardiac failure. Although limit...

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Detalles Bibliográficos
Autores principales: Chamberlain, Jeffrey S., Robb, Melissa, Braun, Serge, Brown, Kristy J., Danos, Olivier, Ganot, Annie, Gonzalez-Alegre, Pedro, Hunter, Nina, McDonald, Craig, Morris, Carl, Tobolowsky, Mark, Wagner, Kathryn R., Ziolkowski, Olivia, Duan, Dongsheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2023
Materias:
Reviews
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10210223/
https://www.ncbi.nlm.nih.gov/pubmed/36694468
http://dx.doi.org/10.1089/hum.2022.190

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