About the Authors
- Karlien Mul
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Roles Supervision, Writing – original draft
Affiliation Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands
- Kate Eichinger
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Roles Conceptualization, Methodology, Writing – review & editing
Affiliation Department of Neurology, University of Rochester Medical Center, Rochester, New York, United States of America
- Man Hung
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Roles Conceptualization, Writing – review & editing
Affiliation Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, United States of America
- Valeria A. Sansone
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Neurorehabilitation Unit, The NeMO Clinical Center, University of Milan, Milan, Italy
- Cynthia Gagnon
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, Université de Sherbrooke, CIUSSS Saguenay–Lac-Saint-Jean, Québec, Canada
- Sub Subramony
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, University of Florida College of Medicine, Gainesville, Florida, United States of America
- Richard H. Roxburgh
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Centre for Brain Research Neurogenetics Clinic, The University of Auckland, Auckland, New Zealand
- Johanna Hamel
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, Division of Neuromuscular Disease, and Department of Pathology and Laboratory Medicine, University of Rochester Medical Center, Rochester, New York, United States of America
- Jeffrey M. Statland
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, University of Kansas Medical Center, Kansas City, Kansas, United States of America
- Bakri Elsheikh
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, Division of Neuromuscular Diseases, The Ohio State University Wexner Medical Center, Columbus, Ohio, United States of America
- Chris Turner
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, The Institute of Neurology, University College London and National Hospital for Neurology and Neurosurgery, Queen Square, London, United Kingdom
- Jacinda Sampson
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology and Neurological Sciences, Stanford University, Stanford, California, United States of America
- Thomas Ragole
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, United States of America
- Emma Matthews
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Atkinson-Morley Neuromuscular Centre, St George’s University Hospitals NHS Foundation Trust, London, United Kingdom
- Benedikt Schoser
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, Friedrich-Baur-Institute, LMU Clinic, Munich, Germany
- Andrea Swenson
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, University of Iowa, Iowa City, Iowa, United States of America
- Chamindra Laverty
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurosciences, University of California, San Diego, California, United States of America
- Perry Shieh
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, University of California, Los Angeles, California, United States of America
- Ericka P. Greene
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Roles Conceptualization, Investigation, Writing – review & editing
Affiliation Houston Methodist Neurological Institute, Houston, Texas, United States of America
- Masanori Takahashi
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Clinical Laboratory and Biomedical Sciences, Division of Clinical Neurophysiology, Osaka University Graduate School of Medicine, Osaka, Japan
- Matthew Wicklund
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Roles Conceptualization, Supervision, Writing – review & editing
Affiliation Department of Neurology, UT Health San Antonio, San Antonio, Texas, United States of America
- Jeanne Dekdebrun
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Roles Conceptualization, Writing – review & editing
Affiliation Department of Neurology, University of Rochester Medical Center, Rochester, New York, United States of America
- Jennifer Raymond
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Roles Conceptualization, Writing – review & editing
Affiliation Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, United States of America
- Erin DeSpain
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Roles Conceptualization, Writing – review & editing
Affiliation Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, United States of America
- Charles A. Thornton
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Roles Conceptualization, Funding acquisition, Supervision, Writing – review & editing
Affiliation Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, United States of America
- Nicholas E. Johnson
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Roles Conceptualization, Funding acquisition, Supervision, Writing – review & editing
* E-mail: nicholas.johnson@vcuhealth.org
Affiliation Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, United States of America
- Myotonic Dystrophy Clinical Research Network
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¶Full membership list can found be found in the ‘Acknowledgments’
Competing Interests
I have read the journal’s policy and the authors of this manuscript have the following competing interests: KM served as a paid consultant for Dyne Therapeutics and Avidity Biosciences. She received research funds from PepGen Inc. KE has received personal compensation for serving on advisory boards and/or as a consultant for Fulcrum Therapeutics, Avidity Biosciences, Roche, and Dyne Therapeutics and TRiNDS. VS serves on Advisory Boards and provides intellectual and scientific activities for Dyne, Avidity, Roche, Biogen, Novartis, Pepgen, Verthex, Arthex, Italfarmaco. CG has consulting agreements with Dyne Therapeutics and Vertex Therapeutics. SS has received prior funding from the NIH, FDA, Friedreich Ataxia Research Alliance, National Ataxia Foundation, Avidity, Vertex, Arthrex, PTC, Reata, Biogen, Retrotope, RENEO, Biohaven, and Fulcrum Therapeutics. SS participates on the board for Reata, Avidity, Biogen, Amicus, Fulcrum, and Lupin. RR has received funding for advisory boards for Roche, Biogen and Larimar, for clinical trials from Dyne, Arrowhead, Edgewise, Reneo, Stealth, Amicus, Roche and for investigator-led research from Biogen and Roche. JH serves on the clinical advisory board for Vertex Therapeutics and Dyne Therapeutics. JMS has received grant funding from the NIH, CDC, MDA, FSHD Society, Friends of FSH Research, and FSHD Canada; and has served on the scientific advisory board or as a consultant for Dyne Therapeutics, Avidity, Fulcrum, Roche, Epic Bio, Armatus, Kate, Alnylam, and MiRecule. BE served as paid consultant for Biogen and Genentech. He received research funds from Biogen, Genentech, NMD Pharma, RaPharma, Alexion, UCB, Pharnext, Avidity, and Celgene. CT has a consulting agreement with Vertex Pharmaceuticals. TR has served as a paid consultant for Alexion, AstraZeneca Rare Disease, and UCB. He has received grant funding from Alexion, AstraZeneca Rare Disease, and argenx SE. BS has served as a paid consultant for Amicus, argenx, Astellas, Avidity, Pepgen, and Sanofi and received speaker honoraria from Amicus, Alexion, Kedrion, and Sanofi. He declares no stocks or shares. CGL has received consulting fees from Novartis, Biogen, Sarepta, Avidity, Dyne, NS Pharma, Takeda, PTC, Solid, and UCB. She is the PI for Sarepta, Avidity, Scholar Rock, Dyne, Biohaven, and Edgewise. MPT has served as a paid consultant for Alexion Pharmaceuticals, argenx, Dyne Therapeutics, Japan Clinical Research Operations, Nippon Shinyaku, and Ono Pharmaceutical, and received speaker honoraria from argenx, Alexion Pharmaceuticals, and UCB Pharma. JD has sered as a paid consultant for Avidity, Dyne, Vertex, Sanofi, PepGen, Arthex, and Lupin. CAT has received royalties and licensing from JAX Laboratories; he has received consulting fees from Sanofi, Biogen, Kate Therapeutics, and Entrada; he has received honoraria from Vertex, Sanofi, and Alnylam, he has participated in an advisory board for Dyne Therapeutics and Pepgen; he serves on board of directors for Myotonic Dystrophy Foundation. NEJ receives royalties from the CCMDHI and the CMTHI. He receives research funds from Novartis, Takeda, PepGen, Sanofi Genzyme, Dyne, Vertex Pharmaceuticals, Fulcrum Therapeutics, AskBio, ML Bio, and Sarepta. He has provided consultation for Arthex, Angle Therapeutics, Juvena, Rgenta, PepGen, AMO Pharma, Takeda, Design, Dyne, AskBio, Avidity, and Vertex Pharmaceuticals. He has equity in Angle Therapeutics, and MyoGene Therapeutics. The other authors have nothing to declare. This does not alter our adherence to PLOS ONE policies on sharing data and materials.