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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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Bibliografiske detaljer
Udgivet i:Ther Adv Neurol Disord
Main Authors: Ruggiero, Lucia, Iodice, Rosa, Esposito, Marcello, Dubbioso, Raffaele, Tozza, Stefano, Vitale, Floriana, Santoro, Lucio, Manganelli, Fiore
Format: Artigo
Sprog:Inglês
Udgivet: SAGE Publications 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6236582/
https://ncbi.nlm.nih.gov/pubmed/30542376
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1756286418809588
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