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Article History

Received: 23 November 2021

Accepted: 30 January 2022

First Online: 14 February 2022

Declarations

:

: Not applicable.

: All authors consented to the publication of this manuscript.

: PM is an employee of the Dutch Medicines Evaluation Board, and the views expressed in this article are his own and may not necessarily reflect the position of, the Medicines Evaluation Board.

: LA is consultant for Orchard, co-investigator for the Metachromatic leukodystrophy trial of Takeda, MEGMA. AB is coinvestigator for a trial in MLD (Shire/Takeda) but receives no personal payment related to this role. JB received honorarium for consulting / ad boards from Omeros, Avrobio, Advanced Clinical, BlueRock, Sanofi, Race Oncology and Medexus (all not related to this topic). FF is an investigator of gene therapy clinical trials for MLD sponsored by Orchard Therapeutics, the license holder of investigational medicinal product arsa-cel. FF has acted as ad hoc consultant for an Orchard Therapeutics and Takeda advisory boards. SabG is speaker honoraria from PTC Therapeutics; advisory boards for Takeda Pharma A/S, bluebird bio GmbH, PTC Therapeutics, Orchard Therapeutics; travel grants from Sanofi Genzyme A/S; Principal Investigator in CT-ORZY-NPC-002 (Orphazyme). SamG received institutional research support from Shire/Takeda. He is advisor and coinvestigator for trials in MLD (Shire/Takeda, Orchard) but receives no personal payment related to this role. Funded by DFG grant GR 4688/2-1. CH is involved in premarketing studies with Idorsia, Sanofi and Protalix, outside the scope of this manuscript. LS is consultant for VICO Therapeutics and receives funding of the German Ministry of Health (BMG grant ZMVI1-2520DAT94E) to LeukoExpert, of the German Ministry of Education and Research (BMBF grant 01GM1907A to Treat-ION and grant 01GM1905A to TreatHSP) and of the European Commission (grant 947588 to the ERN-RND registry). NW is consultant for Passage Bio, Ionis, Orchard, co-investigator for the Metachromatic leukodystrophy trial of Shire/Takeda. She receives research support from Metakids and ZonMW. She is in the scientific advisory board of European Leukodystrophy Association (ELA), Mission Massimo, Yaya foundation. She is editor of Neuropediatrics, member of the editorial boards of Neurology and European Journal of Pediatric Neurology.