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

Advanced Nonsense Mutation Duchenne and Becker Muscular Dystrophy (nmDBMD)
RECRUITING:

PTC has initiated a Phase 2a clinical study of ataluren in patients with advanced nmDBMD who have lost the ability to walk independently. The main goal of the multi-center, open-label trial is to obtain additional information about the long-term safety of ataluren. The study will also evaluate the use of several outcome measures of physical, heart, and lung function in nonambulatory patients.

• Trial Sites
• Frequently Asked Questions
• Clinicaltrials.gov Overview
• Ataluren Overview
• Genetic Testing

Nonsense Mutation Duchenne and Becker Muscular Dystrophy (nmDBMD)
ACTIVE-NOT RECRUITING:

PTC has fully enrolled a Phase 2b clinical study of ataluren in nmDBMD. The trial is a multi-center, randomized, double-blind, placebo-controlled study to determine whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time. Results from this trial are expected to be available in 2010.

• ClinicalTrials.gov Overview

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