PTC Therapeutics has Received a Conditional Approval from the EU for Translarna for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

PTC Therapeutics Recognized for Outstanding Innovation at the 2014 Rare & Orphan Advocacy and Research (ROAR) Awards

In Uncategorized by Cameron

November 14, 2014

PTC Therapeutics, Inc.PTCT, -5.58% received the Outstanding Innovation Award at The 2014 Rare & Orphan Advocacy & Research Awards for Translarna™, the first treatment approved in the European Union for Duchenne muscular dystrophy.

The Rare & Orphan Advocacy & Research (ROAR) Awards were announced during the 5th Annual World Orphan Drug Congress Europe, November 12-14th in Brussels, Belgium. The 2014 ROAR Awards recognize those who have done outstanding work in the field of rare and orphan diseases, honor significant contributions to the development of the sector, and celebrate the great achievements made in the last year.

“We are extremely honored to receive the ROAR award for Outstanding Innovation,” said Stuart Peltz, Ph.D., CEO of PTC Therapeutics, Inc. “This award reflects over a decade and a half of research and development by a dedicated group of scientists and clinicians both at PTC and externally. Translarna™ would not have been possible without the commitment and constant support from patients and their families, advocacy groups, investigators and all the employees at PTC. We are proud to be recognized for the first drug approved for the underlying cause of DMD. ”

The “Outstanding Innovation Award” recognizes those who have demonstrated innovative advancements in rare and orphan drug research, development or advocacy.

The nominees were selected by their peers in the orphan sector and the award recipients were chosen by a panel of judges from the rare disease field.

About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper cellular function. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and in collaborations with leading biopharmaceutical companies. PTC has received conditional marketing authorization in the European Economic Area for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. For more information on the company, please visit our website