Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

IMPORTANCE: An unmet need remains for safe and efficacious treatments for Duchenne muscular dystrophy (DMD). To date, there are limited agents available that address the underlying cause of the disease. OBJECTIVE: To evaluate the safety, tolerability, and efficacy of viltolarsen, a novel antisense o...

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Detalles Bibliográficos
Autores principales: Clemens, Paula R., Rao, Vamshi K., Connolly, Anne M., Harper, Amy D., Mah, Jean K., Smith, Edward C., McDonald, Craig M., Zaidman, Craig M., Morgenroth, Lauren P., Osaki, Hironori, Satou, Youhei, Yamashita, Taishi, Hoffman, Eric P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2020
Materias:
Original Investigation
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7251505/
https://www.ncbi.nlm.nih.gov/pubmed/32453377
http://dx.doi.org/10.1001/jamaneurol.2020.1264

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7251505/
https://www.ncbi.nlm.nih.gov/pubmed/32453377
http://dx.doi.org/10.1001/jamaneurol.2020.1264

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