Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

BACKGROUND: Approximately 13% of boys with Duchenne muscular dystrophy (DMD) have a nonsense mutation in the dystrophin gene, resulting in a premature stop codon in the corresponding mRNA and failure to generate a functional protein. Ataluren (PTC124) enables ribosomal readthrough of premature stop...

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Detalles Bibliográficos
Autores principales: Finkel, Richard S., Flanigan, Kevin M., Wong, Brenda, Bönnemann, Carsten, Sampson, Jacinda, Sweeney, H. Lee, Reha, Allen, Northcutt, Valerie J., Elfring, Gary, Barth, Jay, Peltz, Stuart W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2013
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3859499/
https://www.ncbi.nlm.nih.gov/pubmed/24349052
http://dx.doi.org/10.1371/journal.pone.0081302

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3859499/
https://www.ncbi.nlm.nih.gov/pubmed/24349052
http://dx.doi.org/10.1371/journal.pone.0081302

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