Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial-Reference-Cited by-同舟云学术

Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial

Published:2022-12 Issue:4 Volume:137 Page:309-319
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Hastings Caroline,Liu Benny,Hurst Bryan,Cox Gerald F.,Hrynkow Sharon

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference53 articles.

1. Consensus clinical management guidelines for Niemann-Pick disease type C;Geberhiwot;Orphanet J Rare Dis.,2018

2. High incidence of unrecognized visceral/neurological late-onset Niemann-Pick disease, type C1, predicted by analysis of massively parallel sequencing data sets;Wassif;Genet Med.,2016

3. Recommendations for the detection and diagnosis of Niemann-Pick disease type C: an update;Patterson;Neurol. Clin. Pract.,2017

4. Niemann-Pick disease type C;Vanier;Orphanet J Rare Dis.,2010

5. Recommendations on the diagnosis and management of Niemann-Pick disease type C;N.-C.G.W. Group;Mol. Genet. Metab.,2009

Cited by 19 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Advances in research on potential therapeutic approaches for Niemann-Pick C1 disease;Frontiers in Pharmacology;2024-08-28

2. Evaluation of the landscape of pharmacodynamic biomarkers in Niemann-Pick Disease Type C (NPC);Orphanet Journal of Rare Diseases;2024-07-26

3. Differently increased volumes of multiple brain areas in Npc1 mutant mice following various drug treatments;Frontiers in Neuroanatomy;2024-07-16

4. Overview of clinical, molecular, and therapeutic features of Niemann–Pick disease (types A, B, and C): Focus on therapeutic approaches;Cell Biochemistry and Function;2024-05-07

5. Cyclodextrins and their potential applications for delivering vitamins, iron, and iodine for improving micronutrient status;Drug Delivery and Translational Research;2024-04-26