Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, genetic disease caused by mutations in the DMD gene resulting in an absence of functional dystrophin protein. Viltolarsen, an exon 53 skipping therapy, has been shown to increase endogenous dystrophin levels. Herein, long-term (>2 years) fu...

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Detalles Bibliográficos
Autores principales: Clemens, Paula R., Rao, Vamshi K., Connolly, Anne M., Harper, Amy D., Mah, Jean K., McDonald, Craig M., Smith, Edward C., Zaidman, Craig M., Nakagawa, Tomoyuki, Hoffman, Eric P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2022
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9398057/
https://www.ncbi.nlm.nih.gov/pubmed/35634851
http://dx.doi.org/10.3233/JND-220811

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