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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
Assuntos:
Article
Acesso em linha: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
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados