Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Journal of Inherited Metabolic Disease

Volumn 37, Issue 6, 2014, Pages 979-990

  Hendriksz, Christian J ^a   Burton, Barbara ^b   Fleming, Thomas R ^c   Harmatz, Paul ^d   Hughes, Derralynn ^e   Jones, Simon A ^f   Lin, Shuan Pei ^g   Mengel, Eugen ^h   Scarpa, Maurizio ⁱ   Valayannopoulos, Vassili ^j   Giugliani, Roberto ^k   Slasor, Peter ^l   Lounsbury, Debra ^l   Dummer, Wolfgang ^l  

^a BIRMINGHAM CHILDREN S HOSPITAL NHS FOUNDATION TRUST   (United Kingdom)

^b NORTHWESTERN UNIVERSITY   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

^d CHILDREN S HOSPITAL OAKLAND   (United States)

^e ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

^f UNIVERSITY OF MANCHESTER   (United Kingdom)

^g MACKAY MEMORIAL HOSPITAL   (Taiwan)

^h JOHANNES GUTENBERG UNIVERSITY   (Germany)

ⁱ UNIVERSITY OF PADOVA   (Italy)

^j HÔPITAL NECKER ENFANTS MALADES   (France)

^k HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^l BIOMARIN PHARMACEUTICAL INC   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ELOSULFASE ALFA; KERATAN SULFATE; PLACEBO; GALNS PROTEIN, HUMAN; N ACETYLGALACTOSAMINE 4 SULFATASE;

ADOLESCENT; ADULT; ALLERGIC REACTION; ANAPHYLAXIS; ARTICLE; CHILD; CLIMBING; CONTROLLED STUDY; DENGUE; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; ENDURANCE; ENZYME REPLACEMENT; ENZYMURIA; EXERCISE TEST; FEMALE; FEVER; HEADACHE; HUMAN; HYPERSENSITIVITY; INFUSION RELATED REACTION; INJECTION SITE PAIN; LOWER RESPIRATORY TRACT INFECTION; LUNG FUNCTION TEST; MAJOR CLINICAL STUDY; MALE; MORQUIO SYNDROME; MULTICENTER STUDY; OTITIS MEDIA; OUTCOME ASSESSMENT; PATIENT SAFETY; PHASE 3 CLINICAL TRIAL; PNEUMONIA; RANDOMIZED CONTROLLED TRIAL; URTICARIA; VIRAL UPPER RESPIRATORY TRACT INFECTION; VOMITING; WALKING; CLINICAL TRIAL; MIDDLE AGED; MOTOR ACTIVITY; MUCOPOLYSACCHARIDOSIS IV; PRESCHOOL CHILD; PROCEDURES; RARE DISEASES; TREATMENT OUTCOME; URINE; YOUNG ADULT;

ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; CHONDROITINSULFATASES; DOUBLE-BLIND METHOD; ENZYME REPLACEMENT THERAPY; FEMALE; HUMANS; KERATAN SULFATE; MALE; MIDDLE AGED; MOTOR ACTIVITY; MUCOPOLYSACCHARIDOSIS IV; RARE DISEASES; TREATMENT OUTCOME; WALKING; YOUNG ADULT;

EID: 84911995053     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-014-9715-6     Document Type: Article

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