Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: The utility of animal models of disease in the toxicological evaluation of potential therapeutics

Molecular Genetics and Metabolism

Volumn 114, Issue 2, 2015, Pages 217-225

  Murray, James M ^a   Thompson, Anne Marie ^a,c   Vitsky, Allison ^a,d   Hawes, Michael ^a,e   Chuang, Wei Lien ^a   Pacheco, Joshua ^a   Wilson, Stephen ^b   McPherson, John M ^a   Thurberg, Beth L ^a   Karey, Kenneth P ^a   Andrews, Laura ^a,f  

^a GENZYME CORPORATION   (United States)

^b CHARLES RIVER LABORATORIES   (United States)

^c DARTMOUTH MEDICAL SCHOOL   (United States)

^d PFIZER GLOBAL RESEARCH AND DEVELOPMENT   (United States)

^e Charter Preclinical Services   (United States)

^f ABBVIE INC   (United States)

Author keywords

Acid sphingomyelinase; Animal model; Ceramide; Enzyme replacement therapy; Niemann Pick disease; Toxicity biomarkers

Indexed keywords

CERAMIDE; CHEMOATTRACTANT; GRANULOCYTE COLONY STIMULATING FACTOR; INTERLEUKIN 6; RECOMBINANT ENZYME; RECOMBINANT HUMAN ACID SPHINGOMYELINASE; SPHINGOMYELIN; SPHINGOMYELIN PHOSPHODIESTERASE; UNCLASSIFIED DRUG; CYTOKINE; RECOMBINANT PROTEIN;

ADRENAL HEMORRHAGE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CARDIOGENIC SHOCK; CONTROLLED STUDY; DEATH; DRUG MEGADOSE; FEMALE; HEPATITIS; KERATINOCYTE; LOW DRUG DOSE; LUNG ALVEOLUS MACROPHAGE; MALE; MOUSE; NIEMANN PICK DISEASE; NONHUMAN; PRIORITY JOURNAL; RISK ASSESSMENT; ADRENAL GLAND; ANIMAL; BLOOD; C57BL MOUSE; DEFICIENCY; DISEASE MODEL; DOG; DRUG SCREENING; ENZYME REPLACEMENT; IMMUNOLOGY; INTRAVENOUS DRUG ADMINISTRATION; KNOCKOUT MOUSE; LIVER; LYSOSOME; MACACA FASCICULARIS; METABOLISM; NIEMANN-PICK DISEASE, TYPE A; PATHOLOGY; RAT;

ANIMALIA; CANIS FAMILIARIS; MUS; RATTUS;

ADMINISTRATION, INTRAVENOUS; ADRENAL GLANDS; ANIMALS; CERAMIDES; CYTOKINES; DISEASE MODELS, ANIMAL; DOGS; DRUG EVALUATION, PRECLINICAL; ENZYME REPLACEMENT THERAPY; FEMALE; LIVER; LYSOSOMES; MACACA FASCICULARIS; MALE; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; NIEMANN-PICK DISEASE, TYPE A; RATS; RECOMBINANT PROTEINS; SPHINGOMYELIN PHOSPHODIESTERASE; SPHINGOMYELINS;

EID: 84921790971     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2014.07.005     Document Type: Article

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