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One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?

BACKGROUND: Ataluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials but no report exists about real-life experience. PATIENT/METHODS: we describe three Italian children with nmDMD treated with ataluren for 1 year. Measure...

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Detalles Bibliográficos
Autores principales:	Ruggiero, Lucia, Iodice, Rosa, Esposito, Marcello, Dubbioso, Raffaele, Tozza, Stefano, Vitale, Floriana, Santoro, Lucio, Manganelli, Fiore
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	SAGE Publications 2018
Materias:	Case Series
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6236582/ https://www.ncbi.nlm.nih.gov/pubmed/30542376 http://dx.doi.org/10.1177/1756286418809588

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