Author Contributions: Dr Barohn 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: Statland, Bundy, Wang, Trivedi, Griggs, Barohn, Hanna.
Acquisition of data: Wang, Rayan, Trivedi, Sansone, Salajegheh, Venance, Ciafaloni, Matthews, Meola, Herbelin, Griggs, Barohn.
Analysis and interpretation of data: Statland, Bundy, Trivedi, Salajegheh, Griggs, Barohn.
Drafting of the manuscript: Statland, Bundy, Wang, Salajegheh, Herbelin, Griggs, Barohn.
Critical revision of the manuscript for important intellectual content: Statland, Bundy, Wang, Rayan, Trivedi, Sansone, Salajegheh, Venance, Ciafaloni, Matthews, Meola, Herbelin, Griggs, Barohn, Hanna.
Statistical analysis: Bundy.
Obtained funding: Statland, Wang, Herbelin, Griggs, Barohn.
Administrative, technical, or material support: Bundy, Trivedi, Salajegheh, Herbelin, Griggs.
Study supervision: Wang, Rayan, Trivedi, Sansone, Salajegheh, Venance, Ciafaloni, Matthews, Meola, Herbelin, Griggs, Barohn, Hanna.
Conflict of Interest Disclosures: All authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Dr Venance reported receiving reimbursement for travel from Genzyme and royalties from Wiley Publishing for her role as coeditor of Neurology in Practice: Neuromuscular Disorders. Dr Meola reported being supported in part by AFM (the French Muscular Dystrophy Association). Dr Griggs reported serving as chair of executive committee of the Muscle Study Group, which receives support from pharmaceutical companies; serving on scientific advisory boards for The National Hospital Queen Square and PTC Therapeutics; serving on the editorial boards of NeuroTherapeutics and Current Treatment Opinions in Neurology; receiving royalties from the publication of Andreoli and Carpenter's Cecil Essentials of Medicine, Eighth Edition (W.B. Saunders Company, 2000, 2004, 2007, and 2010) and Cecil Textbook of Medicine, 24th Edition (W.B. Saunders Company, 2000, 2004, 2008, and 2010); and receiving research support from TaroPharma. Dr Barohn reported receiving support from Grifols and Genzyme speakers bureau, and being a consultant for Pfizer, MedImmune, Novartis, and NuFactor. No other authors reported any financial disclosures.
Funding/Support: This study was supported by grants RO1 FD 003454 from the Food and Drug Administration Orphan Products Division and U54 NS059065-05S1 from the National Center for Research Resources and the National Institutes of Health. Additional funding was provided in part by grants UL1 RR 033179 (which is now UL1 TR 000001) from the University of Kansas Medical Center Clinical and Translational Science Awards (CTSA), UL1 RR 024160 from the University of Rochester CTSA, G0601943 from the University College London Medical Research Council Centre, and UL1 RR 024982 NCRR/NIH from the University of Texas Southwestern CTSA. Dr Rayan is a Medical Research Council Clinical Training Fellow. Dr Statland is funded by grant T32 NS07338-20 from the National Institutes of Health Experimental Therapeutics in Neurological Disorders.
Role of the Sponsor: The funding sources had no role in the design and conduct of the study; in the collection, analysis, and interpretation of the data; or in the preparation, review, or approval of the manuscript.
Members of the Consortium for Clinical Investigation of Neurologic Channelopathies: University of Kansas Medical Center—Investigators: Richard J. Barohn, MD (principal investigator, steering committee, manuscript preparation); Yunxia Wang, MD (co-principal investigator, steering committee, manuscript preparation); Jeffrey Statland, MD (steering committee, manuscript preparation); Mazen Dimachkie, MD; Project Manager/Study Coordinator/Clinical Evaluator: Laura Herbelin, CCRP (steering committee, manuscript preparation); JoAnn Miller, BA, CCRP (regulatory program manager); Central Cardiologist: Rhea Pimental, MD.
University of Texas Southwestern Medical Center—Investigators: Jaya Trivedi, MD (manuscript preparation); Study Coordinator: Nina Gorham, CCRP; Clinical Evaluator: Rhonda McLin, PTA, Vivian Gonzales.
Brigham and Women's Hospital—Investigators: Mohammad Kian Salajegheh, MD (steering committee, manuscript preparation); Anthony A. Amato, MD; Study Coordinator: Kristen Roe, BS; Clinical Evaluator: Samantha Chused, MSPT; Essa Kayd, RT.
London Health Science Center (London, Ontario, Canada)—Investigators: Shannon Venance, MD (steering committee, manuscript preparation); Angelica Hahn, MD; Study Coordinators/Clinical Evaluators: Wilma Koopman; Jennifer Verheyden; Ashley Ten Haaf; Christine Piechowicz.
University of Rochester—Investigators: Robert C. Griggs, MD (steering committee, manuscript preparation); Emma Ciafaloni, MD (manuscript preparation); Paul Twydell, DO; Study Coordinators: Katherine Aronson; Kimberly Hart (steering committee); Patricia Smith; Barbara Herr; Kate Eichinger; Shree Pandya; Stephen Bean; Araya Puwanant; Quing Ke; Jill Scheltz; Laura Whitesell.
Institute of Neurology (London, England)—Investigators: Michael Hanna, MD (steering committee, manuscript preparation); Dipa L. Raja Rayan, MRCP (manuscript preparation); Emma Matthews, MRCP (manuscript preparation); Study Coordinators/Clinical Evaluator: Gisela Barreto; Veronica Tan; James Burge; Elizabeth Dewar; Daleen Lopez-Begum; Genetic Testing: Richa Sud; Andrea Haworth; Samuel McCall.
University of Milan (Milan, Italy)—Investigators: Valeria Sansone, MD (manuscript preparation); Giovanni Meola, MD (manuscript preparation); Alice Zanolini, MD; Matteo Ciocca, MD.
University of South Florida (DMCC)—Statistician: Brian Bundy, PhD (steering committee, data analysis, manuscript preparation); Jeffrey Krischer, PhD; Holly Ruhlig; Joseph Gomes; Rachel Richesson; Renee Leduc; Jennifer Pilger.
Data and Safety Monitoring Board Members: John W. Day, MD, PhD (Standford School of Medicine, Stanford, California); Robert T. Leshner, MD (UC San Diego, San Diego, California); Todd A. Mackenzie, PhD (Dartmouth Hitchcock Medical Center, Hanover, New Hampshire); Kathryn Wagner, MD, PhD (The Johns Hopkins School of Medicine, Baltimore, Maryland).
National Institute of Neurological Disorders and Stroke (Bethesda, Maryland) Staff: Joanne C. Odenkirchen, MPH (Clinical Research Project Manager); Randy Stewart, PhD (Program Director).
Additional Contributions: We thank the study participants and their families for their commitment and generosity—they are the impetus for this international collaboration.