Clearance of Hepatic Sphingomyelin by Olipudase Alfa is Associated with Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency

American Journal of Surgical Pathology

Volumn 40, Issue 9, 2016, Pages 1232-1242

  Thurberg, Beth L ^a,b   Wasserstein, Melissa P ^e   Jones, Simon A ^g   Schiano, Thomas D ^f   Cox, Gerald F ^b,c,d   Puga, Ana Cristina ^b  

^a SANOFI   (France)

^b Children's Hospital Boston ^*  (United Kingdom)

^c BOSTON CHILDREN S HOSPITAL   (United States)

^d HARVARD MEDICAL SCHOOL   (United States)

^e CHILDREN S HOSPITAL AT MONTEFIORE   (United States)

^f MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^g UNIVERSITY OF MANCHESTER   (United Kingdom)

Author keywords

enzyme replacement therapy; hyperlipidemia; liver pathology; lysosomal storage disease; Niemann Pick disease type B

Indexed keywords

OLIPUDASE ALFA; RECOMBINANT PROTEIN; SPHINGOMYELIN; SPHINGOMYELIN PHOSPHODIESTERASE;

ADULT; AGED; CLINICAL TRIAL; COMPUTER ASSISTED DIAGNOSIS; DOSE RESPONSE; DRUG EFFECTS; FEMALE; HUMAN; IMMUNOHISTOCHEMISTRY; LIVER; MALE; METABOLISM; MIDDLE AGED; NIEMANN-PICK DISEASE, TYPE A; NIEMANN-PICK DISEASE, TYPE B; PATHOLOGY; PHASE 1 CLINICAL TRIAL; TRANSMISSION ELECTRON MICROSCOPY;

ADULT; AGED; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HUMANS; IMAGE INTERPRETATION, COMPUTER-ASSISTED; IMMUNOHISTOCHEMISTRY; LIVER; MALE; MICROSCOPY, ELECTRON, TRANSMISSION; MIDDLE AGED; NIEMANN-PICK DISEASE, TYPE A; NIEMANN-PICK DISEASE, TYPE B; RECOMBINANT PROTEINS; SPHINGOMYELIN PHOSPHODIESTERASE; SPHINGOMYELINS;

EID: 84976260697     PISSN: 01475185     EISSN: 15320979     Source Type: Journal    
DOI: 10.1097/PAS.0000000000000659     Document Type: Article

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