Off-Label Use of Ataluren in Four Non-ambulatory Patients With Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength

About 15% of Duchenne muscular dystrophy (DMD) cases are caused by point mutations leading to premature stop codons and disrupted synthesis of the dystrophin protein. Stop codon read-through therapy is available with the drug Ataluren (Translarna® by PTC Therapeutics). Following positive results in...

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Detalhes bibliográficos
Publicado no:Front Pediatr
Main Authors: Ebrahimi-Fakhari, Daniel, Dillmann, Ulrich, Flotats-Bastardas, Marina, Poryo, Martin, Abdul-Khaliq, Hashim, Shamdeen, Mohammed Ghiath, Mischo, Bernhard, Zemlin, Michael, Meyer, Sascha
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2018
Assuntos:
Pediatrics
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6206203/
https://ncbi.nlm.nih.gov/pubmed/30406066
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fped.2018.00316
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