TY - RPRT TI - Niemann-Pick C-like endo-lysosomal dysfunction in DHDDS patient cells, a congenital disorder of glycosylation, can be treated with miglustat AU - Best, H. L. AU - Cook, S. R. AU - Waller-Evans, H. AU - Lloyd-Evans, E. PY - 2025 DO - 10.1101/2025.01.08.631939 UR - https://www.biorxiv.org/content/10.1101/2025.01.08.631939v1 ID - 10.1101/2025.01.08.631939 ER -