Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin in a Single Patient with Niemann-Pick C1

Niemann-Pick C, Type 1 (NPC1) is a progressive autosomal recessive neurologic disease caused by defective intracellular cholesterol and lipid trafficking. There are currently no United States Food and Drug Administration approved treatments for NPC1. We undertook a study evaluating the safety, effic...

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Udgivet i:Mol Genet Metab
Main Authors: Maarup, Timothy J., Chen, Agnes H., Porter, Forbes D., Farhat, Nicole Y., Ory, Daniel S., Sidhu, Rohini, Jiang, Xuntian, Dickson, Patricia I.
Format: Artigo
Sprog:Inglês
Udgivet: 2015
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4633280/
https://ncbi.nlm.nih.gov/pubmed/26189084
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2015.07.001
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