This study is a long-term study of ataluren in patients with nonsense mutation Duchenne
Inclusion Criteria -
- Male sex.
- Age ≥5 years.
- Phenotypic evidence of DMD
- Nonsense point mutation in the dystrophin gene
- Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum
of 12 months immediately prior to start of study treatment, with no significant change
in dosage or dosing regimen for a minimum of 3 months immediately prior to start of
- 6MWD ≥150 meters
- Ability to perform timed function tests within 30 seconds
- Willingness and ability to comply with scheduled visits, drug administration plan,
study procedures, laboratory tests, and study restrictions.
- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start
of study treatment.
- Prior or ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
- Prior or ongoing therapy with ataluren.
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Exposure to another investigational drug within 6 months prior to start of study
treatment, or ongoing participation in any interventional clinical trial.
- History of major surgical procedure within 12 weeks prior to start of study treatment,
or expectation of major surgical procedure during the 72-week placebo-controlled
- Requirement for daytime ventilator assistance or any use of invasive mechanical
ventilation via tracheostomy.
- Uncontrolled clinical symptoms and signs of congestive heart failure
- Elevated serum creatinine or cystatin C at screening.