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

Caroline A HastingsCamilo VieiraBenny LiuCyrus BasconClaire GaoRaymond Y WangAlicia CaseySharon Hrynkow

Published in: Orphanet journal of rare diseases (2019)

These expanded access data support the safety and potential benefit of systemic IV administration of HPβCD and provide a platform for two clinical trials to study the effect of intravenous administration of HPβCD in NPC patients.

Keyphrases

clinical trial
end stage renal disease
ejection fraction
high dose
newly diagnosed
chronic kidney disease
young adults
prognostic factors
high throughput
randomized controlled trial
low dose
climate change
ionic liquid
phase ii
middle aged
phase iii
open label
patient reported