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PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy
Date:2/3/2009

encouraged rapid identification of eligible candidates for this important study," said Richard Finkel, MD, attending physician, director of the neuromuscular program, Children's Hospital of Philadelphia. "The completion of this trial enrollment is a striking example of the successes we can achieve when patients, families and the medical community come together to address an area of such high unmet medical need."

"Currently available therapies for nmDMD/BMD may alleviate the symptoms of this progressive debilitating disease, but none address the underlying cause of the disease." said Langdon Miller, MD, chief medical officer of PTC. "Ataluren represents a potential breakthrough as the first treatment designed to enable the formation of functioning dystrophin, the missing protein in patients with nmDMD/BMD."

This is the first pivotal trial for ataluren. In addition, PTC is conducting an open-label, long-term safety study in boys with nmDMD who participated in an early Phase 2a trial. PTC also plans to initiate a second pivotal clinical trial of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) in 2009.

The United States Adopted Names Council (USAN) in conjunction with the World Health Organization, International Nonproprietary Names Committee deemed ataluren's mechanism of action to be significantly novel, warranting creation of a new stem, -luren, for pharmaceuticals that are inducers of ribosomal readthrough of nonsense mutation mRNA stop codons.

"We are pleased to have completed enrollment of our first pivotal study of ataluren in boys and young men with nmDMD/BMD. To have achieved this milestone so far ahead of schedule is a tremendous achievement and we are grateful to the investigators, study coordinators, and the patients and their families for their efforts," said Stuart W. Peltz, PhD, president and CEO, PTC Therapeutics. "We look forward to completing this study ove
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SOURCE PTC Therapeutics, Inc.
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