About the Authors

John T. Kissel

john.kissel@osumc.edu

Affiliation Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio, United States of America

Charles B. Scott

Affiliation CBS Squared, Inc, Fort Washington, Pennsylvania, United States of America

Sandra P. Reyna

Affiliation Department of Neurology, University of Utah School of Medicine, Salt Lake City, Utah, United States of America

Thomas O. Crawford

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

Louise R. Simard

Affiliation Department of Biochemistry and Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada

Kristin J. Krosschell

Affiliation Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, United States of America

Gyula Acsadi

Affiliation Departments of Neurology and Pediatrics, Wayne State University School of Medicine, Detroit, Michigan, United States of America

Bakri Elsheik

Affiliation Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio, United States of America

Mary K. Schroth

Affiliation Department of Pediatrics, University of Wisconsin School of Medicine, Madison, Wisconsin, United States of America

Guy D'Anjou

Affiliation Division of Pediatric Neurology, Hôpital Sainte-Justine Montréal, Montréal, Québec, Canada

Bernard LaSalle

Affiliation Department of Biomedical Informatics, University of Utah School of Medicine, Salt Lake City, Utah, United States of America

Thomas W. Prior

Affiliation Department of Molecular Pathology, Ohio State University, Columbus, Ohio, United States of America

Susan Sorenson

Affiliation Primary Children's Medical Center, Salt Lake City, Utah, United States of America

Jo Anne Maczulski

Affiliation Pediatric Occupational Therapy Services, Chicago, Illinois, United States of America

Mark B. Bromberg

Affiliation Department of Neurology, University of Utah School of Medicine, Salt Lake City, Utah, United States of America

Gary M. Chan

Affiliation Department of Pediatrics, University of Utah, Salt Lake City, Utah, United States of America

Kathryn J. Swoboda

Affiliations Department of Neurology, University of Utah School of Medicine, Salt Lake City, Utah, United States of America, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah, United States of America

for the Project Cure Spinal Muscular Atrophy Investigators' Network

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

Conceived and designed the experiments: JTK SPR TOC LS KJK GA BE MKS GD BL JAM KJS. Performed the experiments: JTK SPR TOC LS KJK GA BE MKS GD BL JAM KJS. Analyzed the data: BL CBS GMC. Contributed reagents/materials/analysis tools: TWP LS BL MBB SR. Wrote the paper: JTK SR TOC KS.