Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

Abstract Background Niemann-Pick Disease Type C (NPC) is an inherited, often fatal neurovisceral lysosomal storage disease characterized by cholesterol accumulation in every cell with few known treatments. Defects in cholesterol transport cause sequestration of unesterified cholesterol within the en...

Full description

Bibliographic Details
Main Authors: Caroline Hastings, Camilo Vieira, Benny Liu, Cyrus Bascon, Claire Gao, Raymond Y. Wang, Alicia Casey, Sharon Hrynkow
Format: Article
Language:English
Published: BMC 2019-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Niemann-pick disease type C
Hydroxypropyl-beta-cyclodextrin
Intravenous administration
Investigational new drug, hepatomegaly, splenomegaly, lung disease
Online Access:http://link.springer.com/article/10.1186/s13023-019-1207-1

Internet

http://link.springer.com/article/10.1186/s13023-019-1207-1

Similar Items