Inclusion Solves Insolubility —Translational Research Cycle from Bedside to Bench and Bench to Bedside for Drug Development Targeting Niemann-Pick Disease Type C—

Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann–Pick Disease Type C Model Mice

(2021) Madoka Fukaura et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Differential mode of cholesterol inclusion with 2‐hydroxypropyl‐cyclodextrins increases safety margin in treatment of Niemann‐Pick disease type C

(2021) Yusei Yamada et al.   BRITISH JOURNAL OF PHARMACOLOGY

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

(2021) Cristin D Davidson et al.   Life Science Alliance

Differential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in Npc1-Null Cells

(2020) Sanzana Hoque et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

In Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β-Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type C

(2019) Nushrat Yasmin et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

(2019) Yuki Maeda et al.   Nanomaterials

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

Predicting the Binding Mode of 2-Hydroxypropyl-β-cyclodextrin to Cholesterol by Means of the MD Simulation and the 3D-RISM-KH Theory

(2018) Yuji Hayashino et al.   JOURNAL OF PHYSICAL CHEMISTRY B

Impact of the Niemann–Pick c1 Gene Mutation on the Total Cellular Glycomics of CHO Cells

(2017) Jun-ichi Furukawa et al.   JOURNAL OF PROTEOME RESEARCH

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

(2017) Daniel S Ory et al.   LANCET

Cyclodextrins and Iatrogenic Hearing Loss: New Drugs with Significant Risk

(2017) Mark A. Crumling et al.   Frontiers in Cellular Neuroscience

In vitro evaluation of 2-hydroxyalkylated β-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C disease

(2016) Yuki Kondo et al.   MOLECULAR GENETICS AND METABOLISM

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

β-Cyclodextrin-threaded Biocleavable Polyrotaxanes Ameliorate Impaired Autophagic Flux in Niemann-Pick Type C Disease

(2015) Atsushi Tamura et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

HPGCD Outperforms HPBCD as a Potential Treatment for Niemann-Pick Disease Type C During Disease Modeling with iPS Cells

(2015) Minami Soga et al.   STEM CELLS

2-Hydroxypropyl-β-Cyclodextrin Acts as a Novel Anticancer Agent

(2015) Masako Yokoo et al.   PLoS One

Population pharmacokinetics of doxapram in low-birth-weight Japanese infants with apnea

(2014) Yuki Ogawa et al.   EUROPEAN JOURNAL OF PEDIATRICS

Reply: Lung toxicity of hydroxyl-β-cyclodextrin infusion

(2013) Muneaki Matsuo et al.   MOLECULAR GENETICS AND METABOLISM

Lung toxicity of hydroxypropyl-β-cyclodextrin infusion

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

Comparative effects of respiratory stimulants on hypoxic neuronal cell injury in SH-SY5Y cells and in hippocampal slice cultures from rat pups

(2013) Kaori Tanaka et al.   PEDIATRICS INTERNATIONAL

Aminophylline, administered at usual doses for rodents in pharmacological studies, induces hippocampal neuronal cell injury under low tidal volume hypoxic conditions in guinea-pigs

(2012) Tomoko Somekawa-Kondo et al.   JOURNAL OF PHARMACY AND PHARMACOLOGY

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

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

Long-Term Parenteral Administration of 2-Hydroxypropyl-β-Cyclodextrin Causes Bone Loss

(2012) Ingrid Kantner et al.   TOXICOLOGIC PATHOLOGY

Evaluation of the vancomycin dosage regimen based on serum creatinine used in the neonatal intensive care unit

(2011) Mitsuru Irikura et al.   PEDIATRICS INTERNATIONAL

Niemann-Pick disease type C

(2010) Marie T Vanier   Orphanet Journal of Rare Diseases

Structure of N-Terminal Domain of NPC1 Reveals Distinct Subdomains for Binding and Transfer of Cholesterol

(2009) Hyock Joo Kwon et al.   CELL

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

Methyl-Beta-Cyclodextrin Improves Fertilizing Ability of C57BL/6 Mouse Sperm after Freezing and Thawing by Facilitating Cholesterol Efflux from the Cells1

(2007) Toru Takeo et al.   BIOLOGY OF REPRODUCTION