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.
Nonsense Mutation Cystic Fibrosis (nmCF)
Active not Recruiting:
This pivotal Phase 3 clinical study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) is a multi-center, randomized, double-blind, placebo-controlled study. The main goals of this study are to understand whether ataluren can improve how nmCF patients feel and function and whether the drug can safely be given over a long period.
Additional information about these ataluren clinical trials can be found at Clinicaltrials.gov or by contacting Patient and Professional Advocacy at
(866) 282-5873, (908) 912-9256 or PatientInfo@ptcbio.com