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Published in: Drugs 14/2014

01-09-2014 | R&D Insight Report

Ataluren: First Global Approval

Author: Nicola J. Ryan

Published in: Drugs | Issue 14/2014

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Abstract

Nonsense mutations are implicated in 5–70 % of individual cases of most inherited diseases, including Duchenne muscular dystrophy (DMD) and cystic fibrosis. Ataluren (Translarna™) is an orally available, small molecule compound that targets nonsense mutations, and is the first drug in its class. Ataluren appears to allow cellular machinery to read through premature stop codons in mRNA, enabling the translation process to produce full-length, functional proteins. This article summarizes the milestones in the development of ataluren leading to its conditional first approval for nonsense mutation DMD.
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Metadata
Title
Ataluren: First Global Approval
Author
Nicola J. Ryan
Publication date
01-09-2014
Publisher
Springer International Publishing
Published in
Drugs / Issue 14/2014
Print ISSN: 0012-6667
Electronic ISSN: 1179-1950
DOI
https://doi.org/10.1007/s40265-014-0287-4

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