CFDB - Cystic Fibrosis DataBase

HTA - Health Technology Assessment Report

Ataluren (Translarna) for CFTR class I nonsense mutation mediated cystic fibrosis (Structured abstract)


cystic fibrosis transmembrane conductance regulator (CFTR) class I nonsense mutation mediated


Ataluren (Translarna; PTC-124) is a 284.24 Da, achiral, 1,2,4-oxadiazole linked to fluorobenzene and benzoic acid rings. It is an orally delivered compound designed to selectively promote ribosomal read-through of premature stop codons, but not normal termination codons

Keywords: PTC124; Ataluren; CFTR Modulators; pharmacological_intervention;