Document is current

Article History

Received: 19 January 2023

Accepted: 5 July 2023

First Online: 13 July 2023

Declarations

:

: The study is conducted following the 1964 Declaration of Helsinki and its later amendments, with approval of the Institutional Review Boards of Clinic for Child Neurology and Psychiatry University of Belgrade (IRB number 1-48/3-2016) and the McGill University Health Center and Montreal Children’s Hospital Research Ethic Boards (11-105-PED and 2019-4972).

: Written informed consents to participate in the research study and written informed consents for publication were obtained from the parents of the affected patients.

: G.B. is/was a consultant for 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 and GM1 gene therapy trials of Passage Bio (2021-present), GM1 natural history study sponsored by the University of Pennsylvania with funding from 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 unrestricted educational grants 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, the MLC Consortium, the H-ABC Clinical Advisory Board 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. L.T.T. currently manages sponsored clinical trials at the site level for Ionis Pharmaceuticals (Alexander disease clinical trial 2021-present), Passage Bio (Krabbe disease and GM1 gangliosidosis clinical trials, 2021-present), and Teva Pharmaceuticals (chronic and episodic migraine clinical trials, 2022-present). Several other sponsored clinical trials are in various stages of the start-up phase. He also manages a GM1 gangliosidosis natural history study sponsored by the University of Pennsylvania with funding from Passage Bio. The other authors declare no competing interests.