The Spinal Muscular Atrophy Foundation and BG Medicine today announced a collaboration to discover plasma biomarkers of drug efficacy for spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers. This project seeks to discover a clinically-useful molecular biomarker, which can then be used to monitor the efficacy of potential drugs in clinical trials.

“We are pleased to be launching this important step in drug development efforts for SMA,” said Karen Chen, Director, Pre-Clinical Research for the Foundation. “The identification of relevant biomarkers is key to the successful development of new therapeutics for this devastating disease. The unbiased discovery approach taken in this project will add substantially to our understanding of disease and drug effects.”
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