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Drugs
Erschienen in: Drugs 14/2014

01.09.2014 | R&D Insight Report

Ataluren: First Global Approval

verfasst von: Nicola J. Ryan

Erschienen in: Drugs | Ausgabe 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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Metadaten
Titel
Ataluren: First Global Approval
verfasst von
Nicola J. Ryan
Publikationsdatum
01.09.2014
Verlag
Springer International Publishing
Erschienen in
Drugs / Ausgabe 14/2014
Print ISSN: 0012-6667
Elektronische ISSN: 1179-1950
DOI
https://doi.org/10.1007/s40265-014-0287-4

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