Key clinical point: Ataluren delays disease progression in patients with nonsense mutation DMD.
Major finding: Age at loss of ambulation was 2.2 years older among patients who received ataluren.
Study details: A post hoc analysis of 210 patients with nonsense mutation DMD from two cohorts.
Disclosures: The Study 019 and CINRG DNHS study groups are sponsored by PTC Therapeutics, and Dr. Bibbiani is an employee of the company.
McDonald C et al. CNS-ICNA 2020. Abstract PL69.