Nonsense Mutation Cystic Fibrosis (nmCF)
RECRUITING:

PTC has initiated a longer-term, Phase 3 clinical study of ataluren in patients with nonsense mutation CF. 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. The trial is a multi-center, randomized, double-blind, placebo-controlled study.

• Trial sites
• Frequently Asked Questions
• ClinicalTrials.gov overview
• Ataluren overview
• Genetic Testing

Nonsense Mutation Hemophilia A&B (nmHA/B)
RECRUITING:

PTC has initiated a Phase 2a clinical trial of ataluren in patients with nonsense mutation hemophilia type A and B (nmHA and nmHB). The trial is a multi-center, open label, dose escalation study. The main goals of the trial are to determine whether treatment with ataluren can result in an increase in Factor VIII or IX levels and whether the drug can safely be given to people with severe hemophilia due to a nonsense mutation.

• Trial sites
• Frequently Asked Questions
• ClinicalTrials.gov Overview
• Ataluren overview
• Genetic Testing

Additional information about these ataluren clinical trials can be found at www.clinicaltrials.gov or by contacting Patient and Professional Advocacy at (866) 282-5873, (908) 912-9256 or PatientInfo@ptcbio.com