WMS 2020 poster: Ataluren delays loss of ambulation and decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD)

Disease progression outcomes in nmDMD patients enrolled in a phase 3 safety study of a mutation-specific therapy in our poster presented at WMS 2020

  • A propensity score-matched analysis of the effect of a mutation-specific DMD therapy and standard of care (SoC; corticosteroids/palliative therapies) on disease progression in patients with nmDMD compared to the effect of SoC alone in patients with DMD
  • Median age at loss of ambulation and decline in forced vital capacity (FVC), as measures of the rate of disease progression, are included

▼ 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 pharmacovigilance@ptcbio.com.

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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