About the Authors

Richard S. Finkel

finkel@email.chop.edu

Affiliation Departments of Neurology and Pediatrics, The Children's Hospital of Philadelphia and The University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America

Thomas O. Crawford

Affiliation Departments of Neurology and Pediatrics, The Johns Hopkins University, Baltimore, Maryland, United States of America

Kathryn J. Swoboda

Affiliation Departments of Neurology and Pediatrics, University of Utah School of Medicine, Salt lake City, Utah, United States of America

Petra Kaufmann

Affiliation Department of Neurology, Columbia University, New York, New York, United States of America

Peter Juhasz

Affiliation BG Medicine, Inc., Waltham, Massachusetts, United States of America

Xiaohong Li

Affiliation BG Medicine, Inc., Waltham, Massachusetts, United States of America

Yu Guo

Affiliation BG Medicine, Inc., Waltham, Massachusetts, United States of America

Rebecca H. Li

Affiliation New England Research Institutes, Inc., Watertown, Massachusetts, United States of America

Felicia Trachtenberg

Affiliation New England Research Institutes, Inc., Watertown, Massachusetts, United States of America

Suzanne J. Forrest

Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

Dione T. Kobayashi

Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

Karen S. Chen

Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

Cynthia L. Joyce

Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

Thomas Plasterer

Affiliation Department of Chemistry and Chemical Biology, Northeastern University, Boston, Massachusetts, United States of America

on behalf of the Pilot Study of Biomarkers for Spinal Muscular Atrophy (BforSMA) Trial Group

Members of the BforSMA Trial Group are listed in List S1

Competing Interests

See below for the full list of competing interests.

Author Contributions

Conceived and designed the experiments: RSF TOC KJS PK YG SJF KSC CLJ. Performed the experiments: RSF TOC KJS PK PJ RHL. Analyzed the data: RSF TOC KJS PK PJ XL RHL FT SJF KSC CLJ TP. Contributed reagents/materials/analysis tools: PJ TP. Wrote the paper: RSF TOC KJS PJ XL YG SJF DTK KSC CLJ TP. Completed statistical analysis: FT XL.