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Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

AIM: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. PATIENTS & METHODS: Study 019 was a long-term Phase III...

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Detalles Bibliográficos
Autores principales:	McDonald, Craig M, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Future Medicine Ltd 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8787621/ https://www.ncbi.nlm.nih.gov/pubmed/34791888 http://dx.doi.org/10.2217/cer-2021-0196

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