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Authors
Lochmüller, Hanns https://orcid.org/0000-0003-2324-8001
Gonorazky, Hernan https://orcid.org/0000-0001-8084-8324
Nigro, Elisa https://orcid.org/0000-0002-5925-2507
Mah, Jean K. https://orcid.org/0000-0002-5795-9955
Alemán, Alberto https://orcid.org/0000-0002-8820-693X
Yaworski, Amanda https://orcid.org/0000-0002-5673-0788
Oskoui, Maryam https://orcid.org/0000-0003-1042-0108
Sbrocchi, Anne Marie https://orcid.org/0009-0005-3682-8293
Selby, Kathryn https://orcid.org/0000-0001-9239-1704
de Vera, Ana https://orcid.org/0009-0004-4183-0155
McAdam, Laura https://orcid.org/0000-0002-4650-7232
Gresko, Ekaterina https://orcid.org/0009-0006-0906-6198
Linden, Aki https://orcid.org/0009-0005-1081-1071
Dutreix, Catherine https://orcid.org/0009-0000-0851-3423
Hoffman, Eric P. https://orcid.org/0000-0001-6470-5139
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Funding
Funding for this research was provided by:
Santhera
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Article History
Received: 11 November 2025
Revised: 13 February 2026
Accepted: 15 February 2026
First Online: 3 March 2026
Declarations
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: Hanns Lochmüller/HL: Consultancy and financial support for research projects and clinical trials: Amplo Biotechnology, AMO Pharma, Argenx, Biogen, Desitin, Fulcrum Therapeutics, Harmony Biosciences, KYE Pharmaceuticals, Milo Biotechnology, Novartis, Pfizer, PTC Therapeutics, Hoffman-La Roche, Sanofi-Genzyme, Santhera Pharmaceuticals, Sarepta, Satellos, Spark Therapeutics, Ultragenyx. Hernan Gonorazky/HG: clinical trial investigator: Pfizer, Santhera Pharmaceuticals; advisor: Kye Pharma, Novartis, Roche, Solid Biosciences; grants: Muscular Dystrophy Canada. Elisa Nigro/EN: no disclosures. Jean K. Mah/JKM: clinical trial investigator: Biogen, Italfarmaco SpA, NS Pharma, Pfizer, PTC Therapeutics, ReveraGen Biopharma, Roche, Santhera Pharmaceuticals, Sarepta Therapeutics. Alberto Alemán/AA: clinical trial investigator: ReveraGen BioPharma, Santhera Pharmaceuticals, Pfizer. Amanda Yaworski/AY: consultant: Novartis, Biogen, Roche. Maryam Oskoui/MO: clinical trial investigator: Novartis Pharmaceuticals, Roche Genentech, Santhera Pharmaceuticals. Anne Marie Sbrocchi/AMS: clinical trial investigator: Ultragenyx, Santhera Pharmaceuticals; consultant: Catalyst, Kyowa Kirin. Kathryn Selby/KS: clinical trial investigator: Santhera Pharmaceuticals. Ana de Vera/AdV, Ekaterina Gresko/EG, Aki Linden/AL, Catherine Dutreix/CD: employees of Santhera Pharmaceuticals. Laura McAdam/LM: Clinical trial investigator: Italfarmaco SpA, Santhera, Anavex Life sciences; consulting: Acadia Pharmaceuticals, Kye therapeutics. Eric P. Hoffman/EPH: stock ownership: ReveraGen BioPharma, AGADA BioSciences; employee: ReveraGen, AGADA BioSciences; clinical trial investigator: Santhera Pharmaceuticals.
: The clinical study protocol and all amendments, informed consent form (ICF), and any other appropriate documents were approved by the Institutional Review Board (IRB)/Independent Ethics Committee (IEC) prior to the start of the study at each of the five recruitment sites (Alberta Children’s Hospital Research Institute, University of Calgary, Calgary, Alberta, Canada; Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada; Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, Canada; Centre for Outcomes Research and Evaluation, Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada; The University of British Columbia, Children's and Women's Health Centre, Vancouver, British Columbia, Canada).
: The parent/legal guardian for each participant provided written consent using the ICF before any study procedures began. Written or verbal assent was also obtained from each participant as required per local regulations.
