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

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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Bibliographic Details
Main Authors: Daniel Ebrahimi-Fakhari, Ulrich Dillmann, Marina Flotats-Bastardas, Martin Poryo, Hashim Abdul-Khaliq, Mohammed Ghiath Shamdeen, Bernhard Mischo, Michael Zemlin, Sascha Meyer
Format: Article
Language:English
Published: Frontiers Media S.A. 2018-10-01
Series:Frontiers in Pediatrics
Subjects:
Translarna
Duchenne muscular dystrophy
DMD
non-ambulatory
nmDMD
stop codon read-through therapy
Online Access:https://www.frontiersin.org/article/10.3389/fped.2018.00316/full

Internet

https://www.frontiersin.org/article/10.3389/fped.2018.00316/full

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