You are Pioneers Once Again


We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community.  CureDuchenne funded PTC Therapeutics back in 2003 and we are proud to have helped advance the development of Translarna.  We look forward to PTC completing their rolling New Drug Application (NDA) with the Food & Drug Administration (FDA).  This is an exciting time for the Duchenne community. We are hopeful to have approved drugs soon for those who live with Duchenne.

Message from PTC

More than 17 years ago PTC Therapeutics was founded and soon began the research that would lead to the development of PTC124, which would become known as ataluren, its generic name, and more recently Translarna, its brand name. We want to thank you for all the support we received at every step of the way. We recently shared the results of ACT DMD, our Phase 3 trial of Translarna in nonsense mutation Duchenne Muscular Dystrophy. We are excited and look forward to submitting the data to the

EMA, as part of our European approval commitment, and to completing our rolling NDA submission in the U.S. As we prepare for potential FDA approval, to complement the approval Translarna has already received in Europe, we want to express our profound gratitude to the entire Duchenne community for helping us get to this important milestone.

This would never have been possible without:

  • the staff and volunteers of Duchenne advocacy groups who believed in PTC from the beginning
  • the clinicians, scientists, physical therapists, site coordinators and nurses who guided us.
  • the patients and families involved in these trials over the years who paved the road for so many

Read more.

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