Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenn...
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
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United States: Public Library of Science
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Language
English
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Publisher
United States: Public Library of Science
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Scope and Contents
Contents
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, leading to production of full-length, functional prote...
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Full title
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
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TN_cdi_plos_journals_1467274319
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1467274319
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ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0081302
