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PTC Therapeutics publicises real-world data Translarna (ataluren)

PTC Therapeutics Announces First Publication of Real-World Data Showing TranslarnaTM (ataluren) Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy.


View the full press release here

  • STRIDE registry analysis shows Translarna preserved ambulation and physical function by years compared with those in CINRG Duchenne Natural History Study, with no new safety signals
  • Trend toward delayed worsening of pulmonary function compared with natural history study


PTC Therapeutics today announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Pulmonary function was also preserved in those treated with Translarna. The analysis, presented in the publication of an interim analysis of preliminary real-world data, compared children treated with Translarna in a real-world setting from the STRIDE registry with a matched cohort in a long-term natural history study, CINRG.

In addition, no new safety signals were observed in the patients treated with Translarna, consistent with what has been shown in previous clinical trials. The interim data have been published in the Journal for Comparative Effectiveness Research. Final data from the STRIDE registry is expected in 2025.