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Article History

Received: 5 January 2023

Accepted: 2 August 2023

First Online: 17 August 2023

Declarations

:

: All methods were carried out in accordance with relevant guidelines and regulations.

: All trials using the core protocol will be ethically reviewed and from all participants consent will be obtained. All studies with VWM patients substantiating this core protocol were approved by the Amsterdam UMC – location VUmc Medical Ethical Committee. All methods were carried out in accordance with relevant guidelines and regulations.

: Not applicable.

: AV: receives funding or in kind support for research from Illumina, Eli Lilly, Takeda, Sana, Affinyia, Sanofi, Passage Bio, Ionis, Homology, Myrtelle, Biogen, Boehringer Ingelheim, Synaptix Bio, without personal compensation.AF: Institutional consultant to Poxel Therapeutics, SwanBio Therapeutics, Adonis Therapeutics. Clinical Trial Site Principal Investigator for Minoryx, and Viking Therapeutics. Coinventor on patent licensed to Ashvattha Therapeutics.BF: The institution of BF has received research support from the National Institutes of Health and the National Ataxia Foundation.EB: co-investigator for trial in Alexander Leukodystrophy (IONIS) with no personal payment.GB: consultant for Passage Bio Inc (2020–2022) and Ionis (2019); site investigator for the Alexander’s disease trial of Ionis (2021-present), Metachromatic leukodystrophy of Shire/Takeda (2020–2021), Krabbe and GM1 gene therapy trials of Passage Bio (2021-present), GM1 natural history study from the University of Pennsylvania sponsored by Passage Bio (2021-present) and Adrenoleukodystrophy/Hematopoietic stem cell transplantation natural history study of Bluebird Bio (2019); site sub-investigator for the MPS II gene therapy trial of Regenxbio (2021-present) and the MPS II clinical trial of Denali (2022-present); received an unrestricted educational grant from Takeda (2021–2022).IKM: advisor for trials in Metachromatic Leukodystrophy and other leukodystrophies (Shire/Takeda, Orchard, PassageBio), without personal payment.JLB: co-investigator for trials in MLD (Shire/Takeda); Krabbe disease (PassageBio); and VWM (Calico), without personal payment. Writer of content for UpToDate on MLD and VWM.MSvdK: consultant for Calico (VWM) and co-investigator for Ionis (Alexander disease trial), without personal payment. She is on patent P112686US00 “therapeutic effects of Guanabenz treatment in vanishing white matter” and on patent P112686CA00 “the use of Guanabenz in the treatment of VWM”, both for the VU University Medical Center, Amsterdam, The Netherlands. She is the initiator and principal investigator of the Guanabenz trial (ExternalRef removed), with permission of the Dutch national ethics committee (CCMO, NL61627.000.18).NIW: advisor and/or co-investigator for trials in Metachromatic Leukodystrophy, Pelizaeus-Merzbacher disease and other leukodystrophies (Shire/Takeda, Orchard, Ionis, PassageBio, VigilNeuro, Sana Biotechnology, Lilly), without personal payment.All other authors (AB, BH, DHS, DS, JB, PSL, EFS-V, RR, HD, PvB, MDS) have nothing to declare.