Fight SMA Provides Grant to PTC Therapeutics, Inc. to Support Research of New Treatments for Spinal Muscular Atrophy

Richmond, Virginia - Fight SMA (www.fightsma.org) has a new partner in its search for a treatment or cure for spinal muscular atrophy (SMA). The international nonprofit organization has established a research and development collaboration with PTC Therapeutics, Inc (PTC) to identify and develop a compound that can be used to treat SMA. SMA is the leading genetic cause of infantile death, yet there currently is no treatment available. PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, and has made great strides toward developing compounds for a variety of genetic disorders including cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).

"This is an exciting opportunity to push toward a treatment for SMA," said Dr. Christian Lorson, PhD, Scientific Director of FightSMA. "The unique genetic context of SMA is well-suited for the type of therapeutic intervention in which PTC Therapeutics specializes, and with the highly skilled scientists at PTC, we are hopeful that great strides can be made towards identifying a drug for this devastating disease."

"We're thrilled that PTC Therapeutics is committed to the fight," said Fight SMA President Martha Slay. "The team at PTC is dedicated and experienced. Theirs is an incredible combination of talent and determination to see these therapies delivered to children and adults suffering with SMA."

PTC Therapeutics, Inc. is also a sponsor of the annual international Fight SMA research conference, The Good Fight. The conference will be held in Washington, DC from April 22nd to the 24th.

About PTC Therapeutics, Inc.
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 of central importance to proper cellular function. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities. PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.

PTC has two product candidates at the clinical stage of development and several preclinical and discovery stage programs. PTC's most advanced product candidate is PTC124, which is currently in Phase 2 clinical trials for cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD) caused by nonsense mutations. PTC299, PTC's anti-angiogenesis drug, is currently in Phase 1 studies in healthy volunteers.

About Fight SMA
Headquartered in Richmond, Virginia, Fight SMA (also known as Andrew's Buddies) is an international nonprofit group dedicated to accelerating research for a treatment or a cure for spinal muscular atrophy (SMA), a neuromuscular disorder that kills more babies than any other genetic disease. For more information on spinal muscular atrophy and Fight SMA, please visit www.fightsma.org. The latest SMA news and research information is available at the Spinal Muscular Atrophy Blog, at www.fightsma.org/blog.


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