CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

Molecular Therapy

Volumn 24, Issue 6, 2016, Pages 1019-1029

  Marshall, John ^a   Sun, Ying ^b,c   Bangari, Dinesh S ^a   Budman, Eva ^a   Park, Hyejung ^a   Nietupski, Jennifer B ^a   Allaire, Amy ^a   Cromwell, Mary A ^a   Wang, Bing ^a   Grabowski, Gregory A ^b,c   Leonard, John P ^a   Cheng, Seng H ^a  

^a SANOFI   (France)

^b CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^c UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD68 ANTIGEN; CERAMIDE GLUCOSYLTRANSFERASE; GLYCOLIPID; VENGLUSTAT; CARBAMIC ACID DERIVATIVE; CONDURITOL EPOXIDE; ENZYME INHIBITOR; GENZ-682452; GLUCOSYLTRANSFERASE; INOSITOL; QUINUCLIDINE DERIVATIVE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BEHAVIORISM; BIOACCUMULATION; CONTROLLED STUDY; DRUG EFFICACY; DRUG MECHANISM; FEMALE; GAIT DISORDER; GAUCHER DISEASE; GLIOSIS; LIFESPAN; LIPID BRAIN LEVEL; LIPID LIVER LEVEL; MOUSE; NONHUMAN; PARESIS; ANALOGS AND DERIVATIVES; ANIMAL; ANTAGONISTS AND INHIBITORS; CENTRAL NERVOUS SYSTEM; CHEMICALLY INDUCED; DISEASE MODEL; DRUG EFFECTS; ENZYMOLOGY; HUMAN; LIVER; LYSOSOME; METABOLISM; ORAL DRUG ADMINISTRATION; TISSUE DISTRIBUTION; TREATMENT OUTCOME;

ADMINISTRATION, ORAL; ANIMALS; CARBAMATES; CENTRAL NERVOUS SYSTEM; DISEASE MODELS, ANIMAL; ENZYME INHIBITORS; GAUCHER DISEASE; GLUCOSYLTRANSFERASES; GLYCOLIPIDS; HUMANS; INOSITOL; LIVER; LYSOSOMES; MICE; QUINUCLIDINES; TISSUE DISTRIBUTION; TREATMENT OUTCOME;

EID: 84962771977     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.53     Document Type: Article

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