Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

BACKGROUND: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations, resulting in absence of functional dystrophin protein. Viltolarsen, an exon 53 skipping therapy, significantly increased dystrophin levels in patients with DMD. Presented here are completed study results of > 4 years o...

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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 2023
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10200237/
https://www.ncbi.nlm.nih.gov/pubmed/37005891
http://dx.doi.org/10.3233/JND-221656

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