Research: Overview

Since its inception in 2003, the SMA Foundation has spent around $150M on basic, translational, and clinical research to accelerate progress towards finding a treatment for SMA. The SMA Foundation drives research through internal and external sponsored research programs in a variety of collaborations. The aim of our research is to expand basic knowledge of the disease and to translate these and other basic findings into therapeutic applications in the clinic. Our efforts are heavily focused on studies and projects that culminate in essential research tools and data that can drive novel drug development forward. Investment by the SMA Foundation has resulted in a large and diverse portfolio of drug discovery assets that are available at minimal or no cost to the biomedical research community. Our major accomplishments include initiation and support of novel drug discovery and biomarker programs and the development of standardized assays.