MDA 2021 poster: Ataluren delays loss of ambulation (LoA) and decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD)

This poster, presented at the virtual 2021 MDA Conference, shows findings from a study comparing the effects of treatment on time to LoA and pulmonary function decline in nmDMD

  • Learn about the background, methods, and baseline demographics of patients in a study to investigate the efficacy of treatment with a mutation-specific DMD therapy + standard of care (SoC) vs SoC alone for nmDMD
  • Explore the results from a comparison of propensity score-matched boys with DMD in Study 019 (n=94) and the CINRG DNHS registry (n=440) comparing age at LoA
  • Review the Kaplan Meier analysis of age at predicted forced vital capacity (FVC) of <60%, <50%, and <1 L in the 2 cohorts

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC at

Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.

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MED-ALL-DMD-2200031 | March 2022
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MED-ALL-CORP-2200029 | December 2022


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