A phase 3 trial of sebelipase alfa in lysosomal acid lipase deficiency

New England Journal of Medicine

Volumn 373, Issue 11, 2015, Pages 1010-1020

  Burton, Barbara K ^a   Balwani, Manisha ^b   Feillet, Francois ^d   Baric, Ivo ^f   Burrow, T Andrew ^g   Grande, Carmen Camarena ^h   Coker, Mahmut ⁱ   Consuelo Sanchez, Alejandra ^k   Deegan, Patrick ^l   Di Rocco, Maja ^m   Enns, Gregory M ^o   Erbe, Richard ^c   Ezgu, Fatih ^j   Ficicioglu, Can ^q   Furuya, Katryn N ^r   Kane, John ^p   Laukaitis, Christina ^s   Mengel, Eugen ^t   Neilan, Edward G ^v   Nightingale, Scott ^x   Peters, Heidi ^y   Scarpa, Maurizio ⁿ   Schwab, K Otfried ^u   Smolka, Vratislav ^aa   Valayannopoulos, Vassili ^e   Wood, Marnie ^z   Goodman, Zachary ^ab   Yang, Yijun ^w   Eckert, Stephen ^w   Rojas Caro, Sandra ^w   Quinn, Anthony G ^w   more..

^a NORTHWESTERN UNIVERSITY   (United States)

^b MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^c CHILDREN S HOSPITAL OF BUFFALO   (United States)

^d CHU NANCY BRABOIS   (France)

^e HÔPITAL NECKER ENFANTS MALADES   (France)

^f KBC Zagreb   (Croatia)

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

^h HOSPITAL UNIVERSITARIO LA PAZ   (Spain)

ⁱ EGE UNIVERSITY SCHOOL OF MEDICINE   (Turkey)

^j GAZI UNIVERSITY FACULTY OF MEDICINE   (Turkey)

^k HOSPITAL INFANTIL DE MÉXICO FEDERICO GÓMEZ   (Mexico)

^l CAMBRIDGE UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^m G GASLINI INSTITUTE   (Italy)

ⁿ UNIVERSITY OF PADOVA   (Italy)

^o STANFORD UNIVERSITY   (United States)

^p UNIVERSITY OF CALIFORNIA   (United States)

^q CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^r NEMOURS ALFRED I DUPONT HOSPITAL FOR CHILDREN   (United States)

^s UNIVERSITY OF ARIZONA   (United States)

^t JOHANNES GUTENBERG UNIVERSITY   (Germany)

^u UNIVERSITY OF FREIBURG   (Germany)

^v BOSTON CHILDREN S HOSPITAL   (United States)

^w Synageva BioPharma Corp   (United States)

^x UNIVERSITY OF NEWCASTLE   (Australia)

^y ROYAL CHILDREN'S HOSPITAL   (Australia)

^z ROYAL BRISBANE AND WOMEN'S HOSPITAL   (Australia)

^aa PALACKÝ UNIVERSITY   (Czech Republic)

^ab INOVA FAIRFAX HOSPITAL   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; GAMMA GLUTAMYLTRANSFERASE; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN CHOLESTEROL; PLACEBO; SEBELIPASE ALFA; TRIACYLGLYCEROL; CHOLESTEROL ESTERASE;

ABDOMINAL PAIN; ADULT; AGED; ARTICLE; ASTHENIA; CHILD; CONSTIPATION; CONTROLLED STUDY; COUGHING; DIARRHEA; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DYSLIPIDEMIA; ENZYME DEFICIENCY; ENZYME REPLACEMENT; EPISTAXIS; FEMALE; FEVER; HEADACHE; HUMAN; IMMUNOGENICITY; LIVER BIOPSY; LIVER CIRRHOSIS; LYSOSOMAL ACID LIPASE DEFICIENCY; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; NAUSEA; OROPHARYNX PAIN; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RHINOPHARYNGITIS; UPPER RESPIRATORY TRACT INFECTION; VOMITING; ADOLESCENT; BIOPSY; BLOOD; CLINICAL TRIAL; DRUG EFFECTS; DYSLIPIDEMIAS; GENETICS; LIVER; MIDDLE AGED; PATHOLOGY; PRESCHOOL CHILD; WOLMAN DISEASE; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; ALANINE TRANSAMINASE; BIOPSY; CHILD; CHILD, PRESCHOOL; CHOLESTEROL, HDL; CHOLESTEROL, LDL; DOUBLE-BLIND METHOD; DYSLIPIDEMIAS; FEMALE; HUMANS; LIVER; MALE; MIDDLE AGED; STEROL ESTERASE; WOLMAN DISEASE; YOUNG ADULT;

EID: 84941312383     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1501365     Document Type: Article

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