Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.

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 codons, lead...

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Bibliographic Details
Main Authors: Richard S Finkel, Kevin M Flanigan, Brenda Wong, Carsten Bönnemann, Jacinda Sampson, H Lee Sweeney, Allen Reha, Valerie J Northcutt, Gary Elfring, Jay Barth, Stuart W Peltz
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2013-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3859499?pdf=render

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http://europepmc.org/articles/PMC3859499?pdf=render

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