Because of our connections to patients and researchers, we are able to advance a comprehensive research program, attacking SMA from all sides.
Basic research investigates the causes and biology of SMA, often revealing new and more effective ways of making drugs. READ MORE
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
Clinical trials test drug candidates for safety and effectiveness. READ MORE
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