In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease

Role of 6- O -α-maltosyl-β-cyclodextrin in lysosomal cholesterol deprivation in Npc1- deficient Chinese hamster ovary cells

(2018) Yasuyo Okada et al.   CARBOHYDRATE RESEARCH

Cholesterol lowering effects of mono-lactose-appended β-cyclodextrin in Niemann–Pick type C disease-like HepG2 cells

(2015) Keiichi Motoyama et al.   Beilstein Journal of Organic Chemistry

Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann–Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease

(2015) Yuta Tanaka et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

(2015) Charles H. Vite et al.   Science Translational Medicine

Small regulators, major consequences - Ca2+ and cholesterol at the endosome-ER interface

(2014) R. van der Kant et al.   JOURNAL OF CELL SCIENCE

In Vitro and In Vivo Study of Gal-OS Self-Assembled Nanoparticles for Liver-Targeting Delivery of Doxorubicin

(2014) Hejian Guo et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

How calcium makes endocytic receptors attractive

(2014) Christian B.F. Andersen et al.   TRENDS IN BIOCHEMICAL SCIENCES

Miglustat: A Review of Its Use in Niemann-Pick Disease Type C

(2013) Katherine A. Lyseng-Williamson   DRUGS

Lung toxicity of hydroxypropyl-β-cyclodextrin infusion

(2013) Yin-Hsiu Chien et al.   MOLECULAR GENETICS AND METABOLISM

Treatment of Niemann–Pick Type C Disease by Histone Deacetylase Inhibitors

(2013) Paul Helquist et al.   Neurotherapeutics

Asialoglycoprotein receptor (ASGPR) as target autoantigen in liver autoimmunity: Lost and found

(2012) Eirini I. Rigopoulou et al.   AUTOIMMUNITY REVIEWS

Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

(2012) Frances M. Platt et al.   JOURNAL OF CELL BIOLOGY

Quantitative role of LAL, NPC2, and NPC1 in lysosomal cholesterol processing defined by genetic and pharmacological manipulations

(2011) Charina M. Ramirez et al.   JOURNAL OF LIPID RESEARCH

Niemann-Pick disease type C

(2010) Marie T Vanier   Orphanet Journal of Rare Diseases

Weekly Cyclodextrin Administration Normalizes Cholesterol Metabolism in Nearly Every Organ of the Niemann-Pick Type C1 Mouse and Markedly Prolongs Life

(2010) Charina M Ramirez et al.   PEDIATRIC RESEARCH

Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid

(2009) Benny Liu et al.   JOURNAL OF LIPID RESEARCH

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

(2009) Cristin D. Davidson et al.   PLoS One

Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport of cholesterol in Niemann-Pick type C cells

(2009) L. Abi-Mosleh et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse

(2009) B. Liu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

(2008) Emyr Lloyd-Evans et al.   NATURE MEDICINE