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PTC Therapeutics Announces Additional $1.6 Million Grant from the Spinal Muscular Atrophy Foundation

Expanded Collaboration Focuses on Discovery of Small-Molecule Drugs for

Spinal Muscular Atrophy

SOUTH PLAINFIELD, N.J., Dec. 6 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms, today announced an expanded research collaboration with the Spinal Muscular Atrophy (SMA) Foundation. The collaboration builds on the existing research agreement to identify and characterize compounds that have the potential to treat SMA by increasing production of the survival motor neuron (SMN) protein, the absence of which causes SMA. Under the terms of the expanded agreement, the SMA Foundation will provide an additional $1.6 million in funding to PTC based upon completion of certain milestones.

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"We are pleased to expand this collaboration with the SMA Foundation," stated Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics. "By applying our proprietary technologies, we have made important progress in our efforts to identify treatments for SMA. This program is another example of the breadth of our approach and we look forward to our continued collaboration with the SMA Foundation as we seek to develop treatments for disorders with high unmet medical need."

"PTC has been an ideal partner in the identification of molecules targeting SMN," noted Meg Winberg, Director of Discovery Research for the SMA Foundation. "We have been impressed with their progress and are pleased to continue our work together to identify and advance potential treatments for SMA."

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a genetic, motor neuron disease characterized by the wasting of skeletal muscles. Caused by progressive degeneration of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA. SMA is often compared to polio because the same spinal cord cells are attacked in each disease. Over time, patients afflicted by this disease continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe.

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 $40 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.

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. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering- Plough.

SOURCE PTC Therapeutics, Inc.
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