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Authors
van Cruchten, Remco T. P.
van As, Daniël
Glennon, Jeffrey C.
van Engelen, Baziel G. M.
‘t Hoen, Peter A. C. http://orcid.org/0000-0003-4450-3112
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Okkersen, K
Jimenez-Moreno, C
Wenninger, S
Daidj, F
Cumming, S
Littleford, R
Monckton, D G
Lochmüller, H
Catt, M
Faber, C G
Hapca, A
Donnan, P T
Gorman, G
Bassez, G
Schoser, B
Knoop, H
Treweek, S
Wansink, Derick G.
Impens, Francis
Gabriels, Ralf
Claeys, Tine
Ravel-Chapuis, Aymeric
Jasmin, Bernard J.
Mahon, Niamh
Nieuwenhuis, Sylvia
Martens, Lennart
Novak, Petr
Furling, Denis
Baak, Arie
Gourdon, Genevieve
MacKenzie, Alex
Martinat, Cecile
Neault, Nafisa
Roos, Andreas
Duchesne, Elise
Salz, Renee
Thompson, Rachel
Baghdoyan, Sandrine
Varghese, Anu Mary
Blom, Paul
Spendiff, Sally
Manta, Alexander
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Funding
Funding for this research was provided by:
HORIZON EUROPE Framework Programme (E-Rare Joint Transnational Call JTC 2018)
Seventh Framework Programme (305697)
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Article History
Received: 14 April 2022
Accepted: 30 September 2022
First Online: 10 November 2022
Declarations
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: The OPTIMISTIC clinical trial (NCT02118779) was conducted in accordance with the Declaration of Helsinki and approved by the medical-ethical scientific committee for human research at each of the four participating clinical centers []. Prior to participation in the OPTIMISTIC study, all participants had provided written informed consent, including the consent for the use of their pseudo-anonymized blood, urine, and DNA samples. Researchers of this study were not able to link the pseudo-anonymized samples to patient identities.
: Not applicable.
: R. van Cruchten reports no disclosures relevant to the manuscript; D. van As reports no disclosures relevant to the manuscript; J.C. Glennon reports no disclosures relevant to the manuscript; B. G. M. van Engelen received fees (to the institution) and non-financial support from Fulcrum Therapeutics, Facio Therapies, and Arrowhead Pharmaceuticals during the conduct of the study. In addition, he received grant support from the FP7 European Union grand OPTIMISTIC, Marigold Foundation Canada, Prinses Beartrix Spierfonds, Spieren voor Spieren, FSHD Stichting, and FSHD Society. He also has an unpaid function as the head of the scientific advisory board for Euro-DyMA. P. A. C. ’t Hoen reports no disclosures relevant to the manuscript.
