Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

<h4>Background</h4>Drisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.<h4>Methods</h4>This 188-week open-label extension of the...

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Main Authors: Nathalie M Goemans, Már Tulinius, Marleen van den Hauwe, Anna-Karin Kroksmark, Gunnar Buyse, Rosamund J Wilson, Judith C van Deutekom, Sjef J de Kimpe, Afrodite Lourbakos, Giles Campion
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2016-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0161955

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https://doi.org/10.1371/journal.pone.0161955

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