About the Authors

Michael Ziemba

Roles Data curation, Formal analysis, Software, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Department of Biomedical Engineering, Binghamton University–State University of New York, Binghamton, NY, United States of America

Molly Barkhouse

Roles Formal analysis, Investigation, Methodology, Project administration, Writing – review & editing

Affiliation AGADA Biosciences, Halifax, Nova Scotia, Canada

Kitipong Uaesoontrachoon

Roles Conceptualization, Formal analysis, Investigation, Methodology, Project administration, Supervision, Writing – original draft, Writing – review & editing

Affiliation AGADA Biosciences, Halifax, Nova Scotia, Canada

Mamta Giri

Roles Data curation, Formal analysis, Visualization, Writing – review & editing

Affiliation Department of Genetics and Genomic Sciences, Mount Sinai Hospital, New York, NY, United States of America

Yetrib Hathout

Roles Data curation, Formal analysis, Writing – review & editing

Affiliation Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University–State University of New York, Binghamton, NY, United States of America

Utkarsh J. Dang

Roles Data curation, Formal analysis, Methodology, Validation, Writing – review & editing

Affiliation Department of Health Outcomes and Administrative Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University–State University of New York, Binghamton, NY, United States of America

Heather Gordish-Dressman

Roles Data curation, Formal analysis, Methodology, Writing – review & editing

Affiliation Center for Translational Sciences, Children’s National Medical Center, Washington, DC, United States of America

Kanneboyina Nagaraju

Roles Conceptualization, Funding acquisition, Methodology, Supervision, Writing – review & editing

nagaraju@binghamton.edu

Affiliations AGADA Biosciences, Halifax, Nova Scotia, Canada, Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University–State University of New York, Binghamton, NY, United States of America

Eric P. Hoffman

Roles Conceptualization, Formal analysis, Funding acquisition, Project administration, Supervision, Visualization, Writing – original draft, Writing – review & editing

Affiliations AGADA Biosciences, Halifax, Nova Scotia, Canada, Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University–State University of New York, Binghamton, NY, United States of America

Competing Interests

Declaration of Interest: KN and EPH hold founder shares and management positions in AGADA Biosciences. MB and KU are employees of AGADA Biosciences. AGADA Biosciences is a contract research organization that received a competitive grant from Foundation to Eradicate Duchenne to perform studies presented in this paper. UD receives consultancy funds from ReveraGen BioPharma. KN and EPH hold founder shares and management positions in ReveraGen BioPharma, the sponsor of vamorolone, a drug in clinical development for Duchenne muscular dystrophy. This does not alter our adherence to PLOS ONE policies on sharing data and materials.