Basic & Translational Research

Understanding disease biology through basic research accelerates the identification of new therapeutic approaches. Translating these basic findings into clinically relevant applications is the next step of the drug development process. The SMA Foundation has invested over $85M in basic and translational research aimed at understanding the biology of SMA pathogenesis, identifying novel therapeutic targets, and developing research tools to systematically and efficiently determine the efficacy of potential treatments. The SMA Foundation's basic/translational research is conducted through collaborations with various research groups including the Harvard Stem Cell Institute and the Columbia University Motor Neuron Center, and through sponsored research agreements with investigators from academic institutions, biotech, and pharmaceutical companies. Through consortia such as the Axon Working Group, the SMA Foundation brings together leading researchers to address critical questions in SMA.