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Ataluren delays disease milestones in patients with nonsense mutation DMD

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.

Citation:

McDonald C et al. CNS-ICNA 2020. Abstract PL69.