Our connections to the spinal muscular atrophy (SMA) community enable us to advance a comprehensive research program that is working to address spinal muscular atrophy from all sides.
Our Research Strategy
Cure SMA’s research strategy for SMA focuses on three main areas:
Basic research investigates the causes and biology of SMA, often revealing new and more effective ways of designing drugs.
Drug discovery converts what we have learned about the causes and biology of SMA into new drug candidates that can be tested in clinical trials.
Clinical trials test new treatments or new applications for approved treatments (e.g., different dosages or different combinations of treatments).