A publicly available tool that contains data from the BforSMA clinical study ( ClinicalTrials.gov, NCT00756821 ), a pilot study to identify candidate biomarkers in blood or urine from a wide range of Spinal Muscular Atrophy (SMA) patients that associate with disease severity. It is hoped that the identification of candidate biomarkers will lead to clinical efficacy and longitudinal natural history studies to verify these markers and enable their use as validated pharmacodynamic markers, longitudinal progression markers, or surrogate endpoint measures in clinical trials.

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