Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

BACKGROUND: Eteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on demonstrated dystrophin production. OBJECTIVE: To report results from PROMOVI, a phase 3, multicenter, open-label study evaluating efficac...

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Detalles Bibliográficos
Autores principales: McDonald, Craig M., Shieh, Perry B., Abdel-Hamid, Hoda Z., Connolly, Anne M., Ciafaloni, Emma, Wagner, Kathryn R., Goemans, Nathalie, Mercuri, Eugenio, Khan, Navid, Koenig, Erica, Malhotra, Jyoti, Zhang, Wenfei, Han, Baoguang, Mendell, Jerry R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8673535/
https://www.ncbi.nlm.nih.gov/pubmed/34120909
http://dx.doi.org/10.3233/JND-210643

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8673535/
https://www.ncbi.nlm.nih.gov/pubmed/34120909
http://dx.doi.org/10.3233/JND-210643

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