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PTC Therapeutics Recognized for Outstanding Innovation at the 2014 Rare & Orphan Advocacy and Research (ROAR) Awards
The Rare & Orphan Advocacy & Research (ROAR) Awards were announced during the 5th Annual World Orphan Drug Congress Europe,
"We are extremely honored to receive the ROAR award for Outstanding Innovation," said
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 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
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