Niemann-Pick disease type C1:  24(S)-hydroxycholesterol pre/post adrabetadex

Name: Niemann-Pick disease type C1: 24(S)-hydroxycholesterol pre/post adrabetadex
Creator: Forbes Porter
Published: 2026-06-22T18:30:12.158Z
Keywords: Clinical Genetics

Porter, Forbes

doi:10.17632/xccvwtr3f2.1

Niemann-Pick disease type C1: 24(S)-hydroxycholesterol pre/post adrabetadex

Published: 22 June 2026| Version 1 | DOI: 10.17632/xccvwtr3f2.1

Contributor:

Forbes Porter

Description

Cerebrospinal fluid 24(S)hydroxycholesterol levels pre and post (48 hr) administration of intrathecal adrabetadex/VTS270?HPBCD in individuals with Niemann-Pick disease type C1

Files

Steps to reproduce

Mol Genet Metab (2025) 146: 109254

Institutions

National Institutes of Health
Maryland, Bethesda

Funders

Eunice Kennedy Shriver National Institute of Child Health and Human Development
National Institutes of Health
Bethesda
Grant ID: ZIA HD008989