Our Strategy

Because of our connections to patients and researchers, we are able to advance a comprehensive research program, attacking SMA from all sides.

Basic Research

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

Drug Discovery

Drug discovery converts basic research ideas into new drug candidates. Learn about our drug discovery process and the different therapeutic approaches we're pursuing. READ MORE 

Therapeutic Approaches 

Clinical Trials

Clinical trials test drug candidates for safety and effectiveness. READ MORE 


Clinical Care

Clinical care research focuses on issues such as breathing or nutrition, with the goal of improving the quality of life for individuals currently living with SMA. READ MORE 

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