RARE Daily

Spinal Muscular Atrophy Foundation and PTC Therapeutics Expand Partnership

March 9, 2021

Rare Daily Staff

The Spinal Muscular Atrophy Foundation and PTC Therapeutics entered into a new collaboration focused on regenerative medicine to further advance scientific research and develop new treatments for SMA and other neuromuscular disorders.

SMA is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. The availability of several disease-modifying therapies for SMA has made regenerative interventions the next frontier in drug discovery and development. The SMA Foundation–PTC partnership will provide funding, managed by the SMA Foundation, to academic institutions and other collaborators to advance foundational research in the area of regenerative medicine.

The Spinal Muscular Atrophy Foundation is a nonprofit organization whose mission is to develop treatments for SMA. Since its inception, the Foundation has funded over $150 million in research and therapeutics development. The Foundation collaborates with leading academic medical centers, the federal government (DoD, NIH, CDC, FDA), and nearly every pharmaceutical company in the world. When the SMA Foundation was established in 2003, there were no treatments for SMA. Due to the foundation’s investments and drug discovery efforts, today there are three FDA-approved drugs.

“In neuromuscular disease, correcting the underlying cause is just the first step. Patients need therapies to help restore lost function. We are excited to expand our partnership with PTC Therapeutics to empower researchers to address this therapeutic gap in neuromuscular disorders with an emphasis on SMA,” said Loren Eng, president of The SMA Foundation. “Our longstanding partnership with PTC lets us harness our research capabilities and PTC’s drug discovery and development expertise and access to core technology platforms, all of which supported the recent approval of Evrysdi in SMA.”

Photo: Loren Eng, president of The SMA Foundation

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