AAN 2023 oral presentation: Safety and efficacy of ataluren in nmDMD patients from Study 041, a phase 3, randomized, double-blind, placebo-controlled trial

This AAN 2023 oral presentation focused on ataluren use for the treatment of nonsense mutation Duchene muscular dystrophy (nmDMD) patients. The symposium detailed the efficacy and safety profile of ataluren in Study 041

  • Appreciate the trial design and patient characteristics in Study 041
  • Explore the efficacy of ataluren in nmDMD across multiple motor endpoints
  • Understand the safety profile of ataluren in nmDMD

▼ 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.

This oral presentation was developed and funded by PTC Therapeutics for AAN 2023.

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