The Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics have entered into a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders.
Recent FDA approvals of several disease modifying therapies for SMA have made regenerative medicine approaches the next frontier in drug discovery and development. These approved treatments correct the underlying cause of these diseases but patients need therapies to help restore lost function.
The SMA Foundation-PTC Therapeutics partnership will address this therapeutic gap by providing funding, managed by the SMA Foundation, to academic institutions and other collaborators to advance research in regenerative medicine. The SMA Foundation and PTC Therapeutics will commit to an initial investment of up to $60M in research to discover and develop promising regenerative treatments for neuromuscular diseases.