Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized, double-blind, placebo-controlled, dose-titration...

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Detalles Bibliográficos
Autores principales: Servais, Laurent, Mercuri, Eugenio, Straub, Volker, Guglieri, Michela, Seferian, Andreea M., Scoto, Mariacristina, Leone, Daniela, Koenig, Erica, Khan, Navid, Dugar, Ashish, Wang, Xiaodong, Han, Baoguang, Wang, Dan, Muntoni, Francesco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Original Papers
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8817703/
https://www.ncbi.nlm.nih.gov/pubmed/34788571
http://dx.doi.org/10.1089/nat.2021.0043

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