Lucerastat, an Iminosugar for Substrate Reduction Therapy: Tolerability, Pharmacodynamics, and Pharmacokinetics in Patients With Fabry Disease on Enzyme Replacement

Nicolas Guérard, Daniel Oder, Peter Nordbeck, +5

Clinical Pharmacology & Therapeutics

Plasma Lysosphingomyelin Demonstrates Great Potential as a Diagnostic Biomarker for Niemann-Pick Disease Type C in a Retrospective Study

Richard W.D. Welford, Marco Garzotti, Charles Marques Lourenço, +7

PLoS ONE

Creutzfeldt-jakob DiseaseNeurological DisorderClinical NeurologyPathologyPlasma Glcsph +21

Enhanced Synthesis of the Oxysterol 24( <i>S</i> ),25-Epoxycholesterol in Macrophages by Inhibitors of 2,3-Oxidosqualene:Lanosterol Cyclase

Andrea H. Rowe, Carmen Argmann, Jane Y. Edwards, +4

Circulation Research

Transport of fluorescent derivatives of fatty acids into cultured human leukemic myeloid cells and their subsequent metabolic utilization

Olivier Morand, Eitan Fibach, Arie Dagan, +1

Biochimica et Biophysica Acta (BBA) - Lipids and Lipid Metabolism

Lipid AnalysisSubsequent Metabolic UtilizationFluorescent DerivativesLipid MetabolismBiochemistry +6

Long‐term survival outcomes of patients with <scp>Niemann‐Pick</scp> disease type C receiving miglustat treatment: A large retrospective observational study

Marc C. Patterson, William S. Garver, R. Giugliani, +10

Journal of Inherited Metabolic Disease

Synthesis and Structure−Activity Studies of Novel Orally Active Non-Terpenoic 2,3-Oxidosqualene Cyclase Inhibitors

Henrietta Dehmlow, Johannes D. Aebi, Synèse Jolidon, +4

Journal of Medicinal Chemistry

The monotopic membrane protein human oxidosqualene cyclase is active as monomer

A. Ruf, Francis Müller, Brigitte D’Arcy, +5

Biochemical and Biophysical Research Communications

Cellular EnzymologyBiochemistryNatural SciencesMetalloproteinMolecular Biology +4

Decrease in Scavenger Receptor Expression in Human Monocyte–Derived Macrophages Treated With Granulocyte Macrophage Colony-Stimulating Factor

Maaike A. van der Kooij, Olivier Morand, H.J.M. Kempen, +1

Arteriosclerosis Thrombosis and Vascular Biology

Immune RegulationImmunologyScavenger ReceptorsImmune SystemInflammation +18

Symptoms and Quality of Life in Patients with Fabry Disease: Results from an International Patient Survey

Olivier Morand, Jack Johnson, Jerry Walter, +5

Advances in Therapy

Induction of lipid storage in cultured leukemic myeloid cells by pyrene-dodecanoic acid

Olivier Morand, Eitan Fibach, Nelly Livni, +1

Biochimica et Biophysica Acta (BBA) - Lipids and Lipid Metabolism

Lipid PreparationLipid StorageBiochemistryNatural SciencesPyrene-dodecanoic Acid +3