Ataluren

Suppresses premature stop mutation

Phase of research

EMA-approved for Other Purpose

How it helps

Restore CFTR Function

General information

Ataluren is a pharmaceutical drug. It was approved in the European Union in 2014 for the treatment of Duchenne muscular dystrophy and it is sold under the name Translarna™. It does not yet have approval from the FDA. Ataluren targets nonsense mutations, where it is able to skip and produce full-length protein. In relation to cystic fibrosis, our AIM tool found the data that ataluren can be beneficial for patients with nonsense mutations who are not treated with tobramycin.

Ataluren on PubChem
Ataluren on DrugBank
Ataluren on Wikipedia

Synonyms

PTC124

Marketed as

Translarna - prescribing information

Dietary sources

N/A

C₁₅H₉FN₂O₃

Drug-Mutation Relation

results for R352Q / G542X

See all data on Ataluren

Treats

Mutation	Link	Tested on	Impact factor	Notes
G542X	A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis	humans	3.48	Effective only in patients not using tobramycin.

Does not treat

Mutation	Link	Tested on	Impact factor	Notes