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

BACKGROUND: 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. METHODS: This 188-week open-label extension of the dose-escalation study assessed the lo...

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Detalles Bibliográficos
Autores principales: Goemans, Nathalie M., Tulinius, Már, van den Hauwe, Marleen, Kroksmark, Anna-Karin, Buyse, Gunnar, Wilson, Rosamund J., van Deutekom, Judith C., de Kimpe, Sjef J., Lourbakos, Afrodite, Campion, Giles
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5010191/
https://www.ncbi.nlm.nih.gov/pubmed/27588424
http://dx.doi.org/10.1371/journal.pone.0161955

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5010191/
https://www.ncbi.nlm.nih.gov/pubmed/27588424
http://dx.doi.org/10.1371/journal.pone.0161955

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