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PTC Therapeutics and Spinal Muscular Atrophy Foundation Announce $8.5 Million Small Molecule Development Collaboration

Project will advance novel platform for treatment of Spinal Muscular Atrophy

SOUTH PLAINFIELD, N.J. and NEW YORK, May 12 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) and the Spinal Muscular Atrophy (SMA) Foundation today announced the expansion of their research collaboration in which the SMA Foundation will provide up to $8.5 million in funding to PTC. This is the second continuation of the collaboration to fund a series of research projects initiated in 2006 to identify and optimize compounds with the potential to treat SMA by increasing production of the survival motor neuron (SMN) protein. The lack of the SMN protein causes SMA. Total funding for the initiative is now $11.7 million.

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"We are very pleased that our collaboration with the SMA Foundation continues to expand and progress," said Stuart W. Peltz, PhD, president and chief executive officer of PTC. "Through this collaboration, we are leveraging our proprietary technologies to identify and develop treatments for a disease with high unmet medical need. Partnerships with venture philanthropy organizations like the SMA Foundation are a core part of our strategy to treat diseases that currently have few or no treatment options."

"The extension of our collaboration with PTC reflects substantial progress to date," said Meg Winberg, PhD, director of discovery research for the SMA Foundation. "We are looking forward to the rapid delivery of significant scientific achievements and, more importantly, an effective treatment for SMA, the most common genetic cause of death among infants and toddlers."

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization 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 of central importance to proper cellular function. PTC's internally-discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. PTC has extensive knowledge of post-transcriptional control processes and has developed proprietary technologies that it applies in its drug discovery activities which have been the basis for collaborations with leading biopharmaceutical companies such as Genzyme, Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's Web site at

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers. Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Over time, patients afflicted by SMA continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA.

About the Spinal Muscular Atrophy Foundation

Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment and cure for spinal muscular atrophy through targeted funding of clinical research and novel drug development efforts. Since its inception, the Foundation has awarded over $60 million in sponsored research agreements. In addition, the Foundation is committed to raising awareness and generating support for increased research efforts in SMA among the leaders of industry and government. For more information on the Spinal Muscular Atrophy Foundation, visit or call (646) 253-7100.

SOURCE PTC Therapeutics, Inc.
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