Effects of sulfatide on peripheral nerves in metachromatic leukodystrophy.

ABSTRACT:

Objective

To evaluate the longitudinal correlations between sulfatide/lysosulfatide levels and central and peripheral nervous system function in children with metachromatic leukodystrophy (MLD) and to explore the impact of intravenous recombinant human arylsulfatase A (rhASA) treatment on myelin turnover.

Methods

A Phase 1/2 study of intravenous rhASA investigated cerebrospinal fluid (CSF) and sural nerve sulfatide levels, 88-item Gross Motor Function Measure (GMFM-88) total score, sensory and motor nerve conduction, brain N-acetylaspartate (NAA) levels, and sural nerve histology in 13 children with MLD. Myelinated and unmyelinated nerves from an untreated MLD mouse model were also analyzed.

Results

CSF sulfatide levels correlated with neither Z-scores for GMFM-88 nor

SUBMITTER: Farah MH 

PROVIDER: S-EPMC10863914 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature