Ataluren has obtained conditional approval for treatment of Duchenne muscular dystrophy and ELX-02 (NB124) is being tested in phase 2 clinical trials in cystic fibrosis patients with nonsense mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (https://www.eloxxpharma.com/clinical-trials/). Here, CFTR is linked to Duchenne muscular dystrophy.