Dr. Stu Peltz from PTC Therapeutics shares his gratitude to the Duchenne community for the contribution families have made to the development of their therapy, Translarna.

Read Dr. Peltz's letter:

You Are Pioneers Once Again! 

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 TranslarnaTM, 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 

YOU are pioneers, YOU have helped to advance the development of Translarna and create a path for companies developing therapies for Duchenne patients. You are owed a debt of gratitude beyond words. We are proud to be partners with you in leading the way toward treatment options in Duchenne and are grateful for the unwavering support of the Duchenne community. 

PTC Therapeutics thanks you! 

Stuart Peltz, PhD 

CEO, PTC Therapeutics 

Click here to download the PDF

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