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Article History

Received: 16 June 2025

Accepted: 22 October 2025

First Online: 28 November 2025

Competing interests

: Authors A.C., S.P., and T.M.D. declare no financial or non-financial competing interests. GB 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 for the Alexander’s disease trial of Ionis (2021-present), Metachromatic leukodystrophy of Shire/Takeda (2020-2021), Krabbe (2021-2023) and GM1 gene therapy trials of Passage Bio (2021-2024), 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), a site sub-investigator for the MPS II gene therapy trial of Regenxbio (2021-present) and the MPS II clinical trial of Denali (2022-present). She has received an unrestricted educational grant from Takeda (2021-2022). She serves on the scientific advisory board of the Pelizaeus-Merzbacher Foundation, the Yaya Foundation Scientific and Clinical Advisory Council and is the Chair of the Medical and Scientific Advisory Board of the United Leukodystrophy Foundation. She is a member of the Vanishing White Matter Consortium, H-ABC Clinical Advisory Board, MLC Clinical Expert Consortium, and the Chair of the POLR3-related (4H) Leukodystrophy Consortium. She is on the editorial boards of Neurology Genetics, Frontiers in Neurology—Neurogenetics, and Journal of Medical Genetics.