About the Authors

Richard S. Finkel

info@ptcbio.com

Affiliations Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America, Nemours Children’s Hospital, Orlando, Florida, United States of America

Kevin M. Flanigan

Current address: Nationwide Children’s Hospital, Columbus, Ohio, United States of America

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

Brenda Wong

Affiliation Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio, United States of America

Carsten Bönnemann

Current address: National Institute of Neurological Disorders and Stroke/NIH Porter Neuroscience Research Center, Bethesda, Maryland, United States of America

Affiliation Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America

Jacinda Sampson

Current address: Columbia University, New York, New York, United States of America

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

H. Lee Sweeney

Affiliation Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Allen Reha

Affiliation PTC Therapeutics, Inc., South Plainfield, New Jersey, United States of America

Valerie J. Northcutt

Affiliation PTC Therapeutics, Inc., South Plainfield, New Jersey, United States of America

Gary Elfring

Affiliation PTC Therapeutics, Inc., South Plainfield, New Jersey, United States of America

Jay Barth

Affiliation PTC Therapeutics, Inc., South Plainfield, New Jersey, United States of America

Stuart W. Peltz

Affiliation PTC Therapeutics, Inc., South Plainfield, New Jersey, United States of America

Competing Interests

AR, VJN, GE, JB, and SWP are employees of PTC Therapeutics, Inc. and hold financial interests in the company. This does not alter the authors’ adherence to all the PLOS ONE policies on sharing data and materials. The following authors work on this study resulted in institutional compensation as a grant awarded to PTC Therapeutics: BW, KMF, RSF, HLS, CB, and JS, PTC employee authors and the PTC Study Group. BW was compensated for travel to meetings for the study. BW and KMF received a consulting fee or honorarium associated with this study. CB’s institution, the Children’s Hospital of Philadelphia, received a consulting fee or honorarium for laboratory supplies and technician time. Regarding financial activities conducted outside of the study presented in this manuscript, the following authors have received compensation for consultancy, or grant for their institution: KMF, RSF, HLS, and JS. HLS also received from various entities, compensation not associated with the submitted work for board membership, royalties, various travel accommodations and meeting expenses. All other authors do not have a relationship with a commercial entity that has an interest in the subject of this manuscript other than the study sponsor.

Author Contributions

Conceived and designed the experiments: RSF KMF BW CB VJN GE SWP. Performed the experiments: RSF KMF BW CB HLS JS. Analyzed the data: RSF KMF BW CB VJN GE SWP AR JB HLS. Contributed reagents/materials/analysis tools: CB HLS. Wrote the paper: RSF KMF BW CB VJN GE SWP AR JB. Performed analysis and interpretation of in vitro dystrophin expression data: HLS. Performed analysis and interpretation of in vivo dystrophin expression data: CB.