Steps Ahead: Optimising physical activity in adults with cystic fibrosis: A pilot randomised trial using wearable technology, goal setting and text message feedback.
cystic fibrosis transmembrane conductance regulator (CFTR) class I nonsense mutation mediated
Interventions
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