Our Strategy

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

Basic research investigates the causes and biology of SMA, often revealing new and more effective ways of designing drugs.

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Drug Discovery

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.

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Clinical Trials

Clinical trials test new treatments or new applications for approved treatments (e.g., different dosages or different combinations of treatments).

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