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Authors
Franken, Linda G.
van Lieshout, Bart
Eugenio, Joshua
Verbeek, Renate
Voermans, Marije
van den Berg, Elske
Bet, Pierre
Mathôt, Ron A. A.
van der Laan, Dennis
Abbink, Truus E. M.
van der Knaap, Marjo S.
Bartelink, Imke H.
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Funding
Funding for this research was provided by:
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
ZonMw (project number 10140261910004)
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Article History
Received: 13 June 2025
Accepted: 6 April 2026
First Online: 19 June 2026
Declarations
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: The authors declare no competing interests.
: The trial is an ongoing open-label single-center study at the University Medical Center Amsterdam in children with DNA-confirmed VWM with onset ≤6 years and disease duration ≤8 years, with approval of the Dutch Central Ethics Committee (CCMO) ( ) and written informed consent from the participant’s parents or legal guardians.
