Poupon-Bejuit, L.
Hughes, M. P.
Liu, W.
Geard, A.
Faour-Slika, N.
Whaler, S.
Massaro, G.
Rahim, A. A.
Funding for this research was provided by:
Action Medical Research (GN2485, GN2485)
Medical Research Council (MR/S036784/1, MR/N026101/1, MR/R025134/1, MR/S036784/1, MR/N026101/1)
AsociaciĆ³n Niemann Pick de Fuenlabrada
Niemann-Pick UK
US National Brain Iron Accumulation Disorders Association
NIHR Great Ormond Street Hospital Biomedical Research Centre (562868)
Wellcome Trust
Wellcome Trust Institutional Strategic Support Fund/UCL Therapeutic Acceleration Support (TAS) Fund
Article History
Received: 28 October 2021
Accepted: 25 July 2022
First Online: 15 August 2022
Competing interests
: The authors declare no competing interests.