Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

Journal of Inherited Metabolic Disease

Volumn 39, Issue 6, 2016, Pages 839-847

  Hendriksz, Christian J ^a   Berger, Kenneth I ^b   Parini, Rossella ^c   AlSayed, Moeenaldeen D ^d   Raiman, Julian ^e   Giugliani, Roberto ^f   Mitchell, John J ^g   Burton, Barbara K ^h   Guelbert, Norberto ⁱ   Stewart, Fiona ^j   Hughes, Derralynn A ^k   Matousek, Robert ^l   Jurecki, Elaina ^l   Decker, Celeste ^l   Harmatz, Paul R ^m  

^a SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^b BELLEVUE HOSPITAL CENTER   (United States)

^c SAN GERARDO HOSPITAL   (Italy)

^d KING FAISAL SPECIALIST HOSPITAL AND RESEARCH CENTER   (Saudi Arabia)

^e HOSPITAL FOR SICK CHILDREN   (Canada)

^f HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^g MONTREAL CHILDREN S HOSPITAL   (Canada)

^h CHILDREN'S MEMORIAL HOSPITAL   (United States)

ⁱ Hospital de Ninos de Cordoba   (Argentina)

^j BELFAST CITY HOSPITAL   (United Kingdom)

^k ROYAL FREE LONDON NHS FOUNDATION TRUST   (United Kingdom)

^l BIOMARIN PHARMACEUTICAL INC   (United States)

^m CHILDREN S HOSPITAL OAKLAND   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ELOSULFASE ALFA; GALNS PROTEIN, HUMAN; N ACETYLGALACTOSAMINE 4 SULFATASE;

ADOLESCENT; ARTICLE; CLINICAL EVALUATION; CLINICAL OUTCOME; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG EFFICACY; ENZYME REPLACEMENT; FOLLOW UP; FORCED EXPIRATORY VOLUME; FORCED VITAL CAPACITY; HUMAN; LUNG FUNCTION TEST; LUNG VENTILATION; MAJOR CLINICAL STUDY; MORQUIO A SYNDROME; MORQUIO SYNDROME; MULTICENTER STUDY; OPEN STUDY; PHASE 3 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL (TOPIC); ADULT; AGED; BREATHING; CHILD; CLINICAL TRIAL; DOUBLE BLIND PROCEDURE; DRUG EFFECTS; FEMALE; LONG TERM CARE; MALE; MIDDLE AGED; PRESCHOOL CHILD; PROCEDURES; RANDOMIZED CONTROLLED TRIAL; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; CHILD; CHILD, PRESCHOOL; CHONDROITINSULFATASES; DOUBLE-BLIND METHOD; ENZYME REPLACEMENT THERAPY; FEMALE; FORCED EXPIRATORY VOLUME; HUMANS; LONG-TERM CARE; MALE; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS IV; RESPIRATION; RESPIRATORY FUNCTION TESTS; YOUNG ADULT;

EID: 84983451273     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-016-9973-6     Document Type: Article

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