Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency

Journal of Hepatology

Volumn 61, Issue 5, 2014, Pages 1135-1142

  Valayannopoulos, Vassili ^a   Malinova, Vera ^b   Honzík, Tomas ^b   Balwani, Manisha ^c   Breen, Catherine ^d   Deegan, Patrick B ^e   Enns, Gregory M ^f   Jones, Simon A ^d   Kane, John P ^g   Stock, Eveline O ^g   Tripuraneni, Radhika ^h   Eckert, Stephen ^h   Schneider, Eugene ^h   Hamilton, Gavin ⁱ   Middleton, Michael S ⁱ   Sirlin, Claude ⁱ   Kessler, Bruce ^j   Bourdon, Christopher ^k   Boyadjiev, Simeon A ^l   Sharma, Reena ^m   Twelves, Chris ⁿ   Whitley, Chester B ^o   Quinn, Anthony G ^h   more..

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b CHARLES UNIVERSITY   (Czech Republic)

^c MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^d ST MARY S HOSPITAL   (United Kingdom)

^e ADDENBROOKE S HOSPITAL   (United Kingdom)

^f STANFORD UNIVERSITY   (United States)

^g UNIVERSITY OF CALIFORNIA   (United States)

^h Synageva BioPharma Corp   (United States)

ⁱ UNIVERSITY OF CALIFORNIA   (United States)

^j Eureka Internal Medicine   (United States)

^k HEALTH SCIENCES NORTH   (Canada)

^l UNIVERSITY OF CALIFORNIA   (United States)

^m SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

ⁿ UNIVERSITY OF LEEDS   (United Kingdom)

^o UNIVERSITY OF MINNESOTA   (United States)

more..

Author keywords

Dyslipidemia; Enzyme replacement; Fatty liver; Hepatomegaly; Lysosomal storage

Indexed keywords

ALANINE AMINOTRANSFERASE; ALKALINE PHOSPHATASE; ASPARTATE AMINOTRANSFERASE; C REACTIVE PROTEIN; FERRITIN; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN CHOLESTEROL; SEBELIPASE ALFA; CHOLESTEROL ESTERASE; LIPA PROTEIN, HUMAN; LIPID; RECOMBINANT PROTEIN;

ABDOMINAL CRAMP; ABDOMINAL PAIN; ADULT; ALLERGIC REACTION; AMINOTRANSFERASE BLOOD LEVEL; ARTICLE; BACKACHE; CHILL; CHOLESTEROL BLOOD LEVEL; CLINICAL ARTICLE; COMMON COLD; DIARRHEA; DISEASE ACTIVITY; DOSE RESPONSE; DRUG SAFETY; DRUG TOLERABILITY; ENZYME DEFICIENCY; FEMALE; FERRITIN BLOOD LEVEL; HEADACHE; HUMAN; HYPEREMIA; IMMUNOGENICITY; INFUSION RELATED REACTION; LIPID BLOOD LEVEL; LIPID LIVER LEVEL; LIVER FUNCTION; LIVER WEIGHT; LYSOSOMAL ACID LIPASE DEFICIENCY; MALE; MIDDLE AGED; NAUSEA; NUCLEAR MAGNETIC RESONANCE IMAGING; PRIORITY JOURNAL; SORE THROAT; UNSPECIFIED SIDE EFFECT; YOUNG ADULT; BLOOD; CLINICAL TRIAL; DEFICIENCY; DRUG ADMINISTRATION; LIVER; MULTICENTER STUDY; PATHOLOGY; WOLMAN DISEASE;

ADULT; ALANINE TRANSAMINASE; ASPARTATE AMINOTRANSFERASES; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; LIPIDS; LIVER; MALE; MIDDLE AGED; RECOMBINANT PROTEINS; STEROL ESTERASE; WOLMAN DISEASE; YOUNG ADULT;

EID: 84921498525     PISSN: 01688278     EISSN: 16000641     Source Type: Journal    
DOI: 10.1016/j.jhep.2014.06.022     Document Type: Article

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