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Article History

Received: 8 October 2021

Accepted: 16 January 2022

First Online: 29 January 2022

Declarations

:

: This manuscript does not report on or involve the use of any animal or human data or tissue.

: This manuscript does not contain any individual persons’ data.

: MK has served on advisory boards for Biogen, Argenx, and CSL Behring, and on the speaker's bureau for Biogen. AP does not have any competing interests to disclose. LA reports grant support from Cure VCP Disease; provides consultancy services for training and quality control through ATOM International for the following companies: Amicus Therapeutics Pty Ltd, Catabasis, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics; reports royalties and other support through Nationwide Children’s Hospital from Sarepta Therapeutics; royalties for licensed technologies; other support through Nationwide Children’s Hospital from Novartis Gene Therapies; advisory board for Biogen. KB has consulted for and/or served as a member of advisory boards for Sanofi—Genzyme, Inventiva Pharma, Amicus Therapeutics, Valerion Therapeutics, and Spark Therapeutics. MJ provides consultancy services for the following companies: ATOM International (covers consultancy services provided to Amicus Therapeutics Pty Ltd, Ascendis Pharma, Biomarin, Catabasis, Faraday, FibroGen, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics, QED Therapeutics Ltd, Reveragen, Sarepta Therapeutics, outside the submitted work. MKJ has received payment for participation on Advisory Boards for F. Hoffman La Roche AG, PTC Therapeutics and fee support for PhD studies from the Jain Foundation, outside the submitted work. NG has participated or is currently participating in clinical trials of anti-dementia drugs sponsored by the following companies: Bristol Myers Squibb, Eli Lilly/Avid Radiopharmaceuticals, Janssen Immunotherapy, Novartis, Pfizer, Wyeth, SNIFF (The Study of Nasal Insulin to Fight Forgetfulness) study, and A4 (The Anti-Amyloid Treatment in Asymptomatic Alzheimer’s Disease) trial. She receives research support from NIH, Tau Consortium, and the Association for Frontotemporal Degeneration. EH does not have any competing interests to disclose. CH has served on advisory boards for Axent Biosciences Inc, Acorda Therapeutics, Amneal Pharmaceuticals, Mitsubishi Tanabe Pharma, and US WorldMeds, and as a consultant for Zywie Bio. SK has served on advisory boards for Alexion Pharmaceuticals, Inc, UCB Pharmaceuticals, Inc, Immunovant, Inc, and Argenx. PM declares the following competing interests: American Heart Association (member of the AHA Career Development Research Grant Committee), California Institute of Regenerative Medicine (member of the Grants Working Group), CareDx Inc. (Site PI for the SHORE Registry), Catabasis Inc. (research grant), Dyne Therapeutics (consultant and member of the DMD Scientific Advisory Board), National Institute of Health (research grants and ad hoc grant reviewer for the NIH SMEP and MOSS Study Sections), Novartis Gene Therapies (member of the Data Monitoring and Safety Committee), and PhaseBio Inc. (research grant and member of the Scientific Advisory Board). SP owns a wellness company for supplements called Wellness with Sujata. GP does not have any competing interests to disclose. SR does not have any competing interests to disclose. BR has served as a consultant for Alexion Pharmaceuticals. WS does not have any competing interests to disclose. AS receives research support from Amylyx Pharmaceuticals, Alexion Pharmaceuticals, ALS Association, Mass General Hospital, NIH/NINDS, and Cytokinetics Inc, and serves on an IDMC for Alexion. TM has served in an advisory capacity for Abbvie, Alexion, Amicus, Argenx, Audentes, Maze Therapeutics, Modis, Momenta, Ra Pharmaceuticals, Sanofi-Genzyme, Sarepta, Spark Therapeutics, UCB, and Ultragenyx. He serves on the speaker’s bureau for Sanofi-Genzyme. Dr. Mozaffar serves on the medical advisory board for the Myositis Association, Neuromuscular Disease Foundation, Myasthenia Gravis Foundation of California and Myasthenia Gravis Foundation of America. Dr. Mozaffar receives research funding from the Myositis Association, the Muscular Dystrophy Association, the National Institutes for Health and from the following sponsors: Alexion, Amicus, Argenx, Audentes, Bristol-Myers-Squib, Cartesian Therapeutics, Grifols, Momenta, Ra Pharmaceuticals, Sanofi-Genzyme, Spark Therapeutics, UCB, and Valerion. He serves on the data safety monitoring board for Acceleron, Avexis, Sarepta, and the NIH. CW has served on advisory boards for Acceleron, Sarepta, Abata, and Orphazyme, and as a consultant for Sarepta. VK does not have any competing interests to disclose.