FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts

The sphingosine-1-phosphate analogue, FTY-720, promotes the proliferation of embryonic neural stem cells, enhances hippocampal neurogenesis and learning and memory abilities in adult mice

(2016) Yili Sun et al.   BRITISH JOURNAL OF PHARMACOLOGY

Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model

(2016) Md. Suhail Alam et al.   Science Translational Medicine

Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target

(2016) A Cougnoux et al.   Cell Death & Disease

Effect of FTY720 (fingolimod) on graft survival in renal transplant recipients: a systematic review protocol: Table 1

(2016) Reza Gholamnezhadjafari et al.   BMJ Open

Immune dysfunction in Niemann-Pick disease type C

(2015) Nick Platt et al.   JOURNAL OF NEUROCHEMISTRY

Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann–Pick C1

(2015) Timothy J. Maarup et al.   MOLECULAR GENETICS AND METABOLISM

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

Elevated Nuclear and Cytoplasmic FTY720-Phosphate in Mouse Embryonic Fibroblasts Suggests the Potential for Multiple Mechanisms in FTY720-Induced Neural Tube Defects

(2015) Nicole M. Gardner et al.   TOXICOLOGICAL SCIENCES

Intracellular sphingosine releases calcium from lysosomes

(2015) Doris Höglinger et al.   eLife

Fingolimod induces neurogenesis in adult mouse hippocampus and improves contextual fear memory

(2015) P Efstathopoulos et al.   Translational Psychiatry

The phosphorylated prodrug FTY720 is a histone deacetylase inhibitor that reactivates ERα expression and enhances hormonal therapy for breast cancer

(2015) N C Hait et al.   Oncogenesis

Inhibiting epigenetic enzymes to improve atherogenic macrophage functions

(2014) Jan Van den Bossche et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Collaborative Development of 2-Hydroxypropyl-β-Cyclodextrin for the Treatment of Niemann-Pick Type C1 Disease

(2014) Elizabeth Ottinger et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Active, phosphorylated fingolimod inhibits histone deacetylases and facilitates fear extinction memory

(2014) Nitai C Hait et al.   NATURE NEUROSCIENCE

The potential of histone deacetylase inhibitors in Niemann - Pick type C disease

(2013) Michael Maceyka et al.   FEBS Journal

Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling

(2013) Martin Fan et al.   JOURNAL OF LIPID RESEARCH

Efficacy of Miglustat in Niemann–Pick C disease: A single centre experience

(2013) Virginia Maria Ginocchio et al.   MOLECULAR GENETICS AND METABOLISM

SAHA Enhances Synaptic Function and Plasticity In Vitro but Has Limited Brain Availability In Vivo and Does Not Impact Cognition

(2013) Jesse E. Hanson et al.   PLoS One

Ryanodine receptor antagonists adapt NPC1 proteostasis to ameliorate lipid storage in Niemann–Pick type C disease fibroblasts

(2012) Ting Yu et al.   HUMAN MOLECULAR GENETICS

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

Hearing Loss and Hair Cell Death in Mice Given the Cholesterol-Chelating Agent Hydroxypropyl-β-Cyclodextrin

(2012) Mark A. Crumling et al.   PLoS One

Quantitative Proteomic Analysis of Niemann-Pick Disease, Type C1 Cerebellum Identifies Protein Biomarkers and Provides Pathological Insight

(2012) Stephanie M. Cologna et al.   PLoS One

BZM055, an Iodinated Radiotracer Candidate for PET and SPECT Imaging of Myelin and FTY720 Brain Distribution

(2011) Emmanuelle Briard et al.   ChemMedChem

An “Exacerbate-reverse” Strategy in Yeast Identifies Histone Deacetylase Inhibition as a Correction for Cholesterol and Sphingolipid Transport Defects in Human Niemann-Pick Type C Disease

(2011) Andrew B. Munkacsi et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts

(2011) N. H. Pipalia et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Amino acid substitution in NPC1 that abolishes cholesterol binding reproduces phenotype of complete NPC1 deficiency in mice

(2011) X. Xie et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification of Surface Residues on Niemann-Pick C2 Essential for Hydrophobic Handoff of Cholesterol to NPC1 in Lysosomes

(2010) Michael L. Wang et al.   Cell Metabolism

FTY720 Stimulates 27-Hydroxycholesterol Production and Confers Atheroprotective Effects in Human Primary Macrophages

(2010) Tomas Blom et al.   CIRCULATION RESEARCH

Fingolimod (FTY720): discovery and development of an oral drug to treat multiple sclerosis

(2010) Volker Brinkmann et al.   NATURE REVIEWS DRUG DISCOVERY

Lipids on Trial: The Search for the Offending Metabolite in Niemann-Pick type C Disease

(2010) Emyr Lloyd-Evans et al.   TRAFFIC

Targeting the correct HDAC(s) to treat cognitive disorders

(2010) André Fischer et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate

(2009) Lutz Garbes et al.   HUMAN MOLECULAR GENETICS

Ceramide Synthesis Is Modulated by the Sphingosine Analog FTY720 via a Mixture of Uncompetitive and Noncompetitive Inhibition in an Acyl-CoA Chain Length-de pend ent Manner

(2009) Sujoy Lahiri et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

FTY720 Inhibits Ceramide Synthases and Up-regulates Dihydrosphingosine 1-Phosphate Formation in Human Lung Endothelial Cells

(2009) Evgeny V. Berdyshev et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

Regulation of Histone Acetylation in the Nucleus by Sphingosine-1-Phosphate

(2009) N. C. Hait et al.   SCIENCE

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

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

Therapeutic application of histone deacetylase inhibitors for central nervous system disorders

(2008) Aleksey G. Kazantsev et al.   NATURE REVIEWS DRUG DISCOVERY

The many roles of histone deacetylases in development and physiology: implications for disease and therapy

(2008) Michael Haberland et al.   NATURE REVIEWS GENETICS