About the Authors

Thomas O. Crawford

tcrawfo@jhmi.edu

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

Sergey V. Paushkin

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

Suzanne J. Forrest

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

Richard S. Finkel

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

Petra Kaufmann

Affiliation Department of Neurology, Columbia University, New York, New York, 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

Danilo Tiziano

Affiliation Instituto di Genetica Medica, Università Cattolica S. Cuore, Roma, Italy

Rosa Lomastro

Affiliation Instituto di Genetica Medica, Università Cattolica S. Cuore, Roma, Italy

Rebecca H. Li

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

Felicia L. Trachtenberg

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

Thomas Plasterer

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

Karen S. Chen

Affiliation Spinal Muscular Atrophy Foundation, New York, New York, 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: TOC SVP DTK SJF CLJ RSF PK KJS RL KSC. Performed the experiments: TOC DTK RSF PK KJS RHL. Analyzed the data: TOC SVP SJF CLJ RSF PK KJS DT RHL FLT TP KSC. Contributed reagents/materials/analysis tools: DT TP. Wrote the paper: TOC SVP DTK SJF CLJ RSF KJS TP KSC.