Our lead product candidate is ataluren for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. Following is a list of ongoing ataluren clinical trials in patients with nonsense mutation genetic disorders. Read the ataluren overview to learn more about ataluren.
Ataluren for nmDMD: Confirmatory Phase 3 clinical trial complete
Ataluren for nmCF: Phase 3 trial complete; Confirmatory Phase 3 clinical trial initiated