Evaluation of Two Liver Treatment Strategies in a Mouse Model of Niemann–Pick-Disease Type C1

Increased Regenerative Capacity of the Olfactory Epithelium in Niemann–Pick Disease Type C1

(2017) Anja Meyer et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

Defects in the retina of Niemann-pick type C 1 mutant mice

(2014) Xin Yan et al.   BMC NEUROSCIENCE

Reduced cerebellar neurodegeneration after combined therapy with cyclodextrin/allopregnanolone and miglustat in NPC1: A mouse model of Niemann-Pick type C1 disease

(2014) Fabian Maass et al.   JOURNAL OF NEUROSCIENCE RESEARCH

The C57BL/6J Niemann–Pick C1 mouse model with decreased gene dosage has impaired glucose tolerance independent of body weight

(2013) David Jelinek et al.   GENE

Combined therapy with cyclodextrin/allopregnanolone and miglustat improves motor but not cognitive functions in Niemann–Pick Type C1 mice

(2013) M. Hovakimyan et al.   NEUROSCIENCE

Pre-Symptomatic Activation of Antioxidant Responses and Alterations in Glucose and Pyruvate Metabolism in Niemann-Pick Type C1-Deficient Murine Brain

(2013) Barry E. Kennedy et al.   PLoS One

Oxidative stress in Niemann-Pick type C patients: a protective role of N-butyl-deoxynojirimycin therapy

(2012) Graziela S. Ribas et al.   INTERNATIONAL JOURNAL OF DEVELOPMENTAL NEUROSCIENCE

Cyclodextrin mediates rapid changes in lipid balance inNpc1−/−mice without carrying cholesterol through the bloodstream

(2012) Anna M. Taylor et al.   JOURNAL OF LIPID RESEARCH

Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update

(2012) Marc C. Patterson et al.   MOLECULAR GENETICS AND METABOLISM

Effects of cyclodextrin in two patients with Niemann–Pick Type C disease

(2012) Muneaki Matsuo et al.   MOLECULAR GENETICS AND METABOLISM

Liver X Receptor: an oxysterol sensor and a major player in the control of lipogenesis

(2011) S. Ducheix et al.   CHEMISTRY AND PHYSICS OF LIPIDS

Unesterified Cholesterol Accumulation in Late Endosomes/Lysosomes Causes Neurodegeneration and Is Prevented by Driving Cholesterol Export from This Compartment

(2011) A. Aqul et al.   JOURNAL OF NEUROSCIENCE

Regulation of Cholesterol and Fatty Acid Synthesis

(2011) J. Ye et al.   Cold Spring Harbor Perspectives in Biology

Recovery from liver disease in a Niemann-Pick type C mouse model

(2010) Naomi L. Sayre et al.   JOURNAL OF LIPID RESEARCH

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

Acyl-coenzyme A:cholesterol acyltransferases

(2009) Ta-Yuan Chang et al.   AMERICAN JOURNAL OF PHYSIOLOGY-ENDOCRINOLOGY AND METABOLISM

On the mechanism for PPAR agonists to enhance ABCA1 gene expression

(2009) Masaki Ogata et al.   ATHEROSCLEROSIS

Effects of Fibrate Drugs on Expression of ABCA1 and HDL Biogenesis in Hepatocytes

(2009) Mohammad Anwar Hossain et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY

The National Niemann-Pick Type C1 Disease Database: correlation of lipid profiles, mutations, and biochemical phenotypes

(2009) William S. Garver et al.   JOURNAL OF LIPID 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

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

Lipotoxicity in nonalcoholic fatty liver disease: not all lipids are created equal

(2009) Naim Alkhouri et al.   Expert Review of Gastroenterology & Hepatology

Substrate reduction therapy

(2008) Frances M Platt et al.   ACTA PAEDIATRICA