Beerepoot, Shanice http://orcid.org/0000-0003-2945-6784
Boelens, Jaap Jan http://orcid.org/0000-0003-2232-6952
Lindemans, Caroline http://orcid.org/0000-0002-3984-5620
de Witte, Moniek A. http://orcid.org/0000-0001-7470-2994
Nierkens, Stefan http://orcid.org/0000-0003-3406-817X
Vrancken, Alexander F. J. E. http://orcid.org/0000-0003-4649-6534
van der Knaap, Marjo S. http://orcid.org/0000-0001-8912-0954
Bugiani, Marianna http://orcid.org/0000-0001-7689-3042
Wolf, Nicole I. http://orcid.org/0000-0003-1721-0728
Funding for this research was provided by:
Stichting Metakids (2017-073)
Article History
Received: 1 December 2023
Revised: 5 March 2024
Accepted: 10 March 2024
First Online: 2 April 2024
Declarations
:
: SB, MAW, SN, and AFJEV report no competing interests. JJB consulting Bluerock, Sanofi, Sobi, Omeros, SmartImmune, Immusoft, Advanced Clinical. CL is advisor in Orchard and Bluebird Bio expertise panels. MB is co-investigator to Cerevance and Covance, both within projects or remyelinating compounds and without personal payment. MSvdK is co-investigator to Ionis (trial in Alexander disease); and consultant to Calico, all without personal payment. She has a patent P112686CA00, therapeutic effects of Guanabenz treatment in vanishing white matter, to VU University Medical Center. NIW is advisor and/or co-investigator for trials in Metachromatic Leukodystrophy and other leukodystrophies (Shire/Takeda, Orchard, Ionis, PassageBio, VigilNeuro, Sana Biotech), without personal payment.
: This study was approved by the Institutional Review Board of Amsterdam UMC, and patients/guardians gave written informed consent.