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Authors
van Voorst, Romy J.
Schoenmakers, Daphne H.
van Beelen, Irene
Gavazzi, Francesco
Chapleau, Alexandra
Vanderver, Adeline
Bernard, Geneviève
Krägeloh-Mann, Ingeborg
van der Knaap, Marjo S. https://orcid.org/0000-0001-8912-0954
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Funding
Funding for this research was provided by:
Netherlands Organization of Scientific Research
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Article History
Received: 10 February 2025
Accepted: 7 August 2025
First Online: 26 August 2025
Declarations
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: The study was approved by the medical ethical com. mittee (Medisch Ethische Toetsingscommissie VU medical center, reference number 2018.588 for the VWM registry and phenotyping patients with VWM, and reference number 2021.0798 for investigating unaffected siblings).
: All authors consented to the publication of this manuscript.
: Marjo S. van der Knaap is co-investigator for Ionis (Alexander disease trial) and Calico / AbbVie (vanishing white matter trials). She is on patent P112686US00 “therapeutic effects of Guanabenz treatment in vanishing white matter” and patent P112686CA00 “the use of Guanabenz in the treatment of vanishing white matter”, for the Amsterdam University Medical Center, Amsterdam, The Netherlands. She is the initiator and principal investigator of the Guanabenz trial for vanishing white matter (). Geneviève Bernard is/was a consultant for Calico (2023-present), Orchard Therapeutics (2023), Passage Bio Inc (2020–2022), and Ionis (2019). She is/was a site investigator/sub-investigator for studies from Ionis (2021-present), Denali (2022-present), Regenxbio (2021-present), Shire/Takeda (2020–2021), Passage Bio (2021–2024), and Bluebird Bio (2019). She has received unrestricted educational grants from Takeda (2021–2022). She is the initiator and principal investigator of “Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/ Vanishing White Matter Disease” (2025–10780 (CLE-ABBV-CLS-7262).
