Blau lab receives grant from the Spinal Muscular Atrophy Foundation and the Stanford Innovative Medicines Accelerator
Gene therapy has shown remarkable promise for the successful treatment of the root cause of spinal muscular atrophy (SMA). However, combined therapeutic approaches that treat all aspects of SMA, including regenerating muscle, are still urgently needed. In this project, jointly funded by the SMA Foundation and the Stanford IMA, the Blau lab will test their hypothesis that 15-PGDH inhibition will synergize with an available therapy that restores SMN expression to increase neuromuscular junctions and improve muscle function.