The Spinal Muscular Atrophy Foundation today announced that they have initiated a research and development collaboration with PTC Therapeutics, Inc. (PTC). The collaboration is designed to leverage PTC’s proprietary Gene Expression Modulation by Smallmolecules (GEMS) technology to identify and develop new small molecule therapeutics for use in the treatment or prevention of spinal muscular atrophy (SMA), a neuromuscular disease and the leading genetic cause of death among infants and toddlers. Under the terms of the agreement, the SMA Foundation will provide up to $1.6 million in funding to PTC based upon completion of certain milestones.

“PTC has demonstrated a strong commitment to SMA and other pediatric orphan diseases,” said Loren Eng, President of the SMA Foundation. “We believe PTC’s scientific assets and proven accomplishments make them an ideal partner in our efforts to identify treatments for SMA.”
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