Accepted for Publication: October 28, 2011.
Author Contributions: Dr Sadun had full access to all of the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis. Study concept and design: Sadun, Chicani, Thoolen, Shrader, Kubis, Carelli, and Miller. Acquisition of data: Sadun, Chicani, Ross-Cisneros, Kubis, and Carelli. Analysis and interpretation of data: Sadun, Chicani, Barboni, Carelli, and Miller. Drafting of the manuscript: Sadun, Chicani, Kubis, and Carelli. Critical revision of the manuscript for important intellectual content: Sadun, Chicani, Ross-Cisneros, Barboni, Thoolen, Shrader, and Carelli. Obtained funding: Sadun, Chicani, Carelli, and Miller. Administrative, technical, and material support: Sadun, Chicani, Ross-Cisneros, Thoolen, Shrader, and Miller. Study supervision: Sadun, Barboni, and Kubis.
Financial Disclosure: Dr Sadun reports receiving unrestricted research funds from Edison Pharmaceuticals Inc. Dr Chicani and Mr Ross Cisneros report that they receive partial salary support from Edison Pharmaceuticals Inc. Dr Carelli reports that he is a consultant for Edison Pharmaceuticals Inc. Drs Shrader, Thoolen, and Miller are employees of Edison Pharmaceuticals Inc.
Role of the Sponsor: The sponsor of the study had a role in the development of the emergency treatment protocol, and writing background information related to EPI-743 and inherited mitochondrial disease clinical trials, but not in final study design, data collection, data analysis, or data interpretation. The corresponding author, Dr Sadun, had full access to all study data and had final responsibility for the decision to submit this paper for publication.
Disclaimer: The views expressed in this article are those of the authors and do not necessarily reflect the official policy or position of the Department of the Navy, Department of Defense, or the US Government.
Additional Contributions: Shellee Rockwell and Richard Cornwell provided clinical care to our patients.