Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial

Molecular Genetics and Metabolism

Volumn 114, Issue 2, 2015, Pages 178-185

  Hendriksz, Christian J ^a,s   Giugliani, Roberto ^b   Harmatz, Paul ^c   Mengel, Eugen ^d   Guffon, Nathalie ^e   Valayannopoulos, Vassili ^f   Parini, Rossella ^g   Hughes, Derralynn A ^h   Pastores, Gregory M ^i,ak   Lau, Heather A ⁱ   Al Sayed, Moeenaldeen D ^j   Raiman, Julian ^k   Ben Omran, Tawfeg ^l   Bober, Michael B ^m   Burton, Barbara K ⁿ   Cleary, Maureen A ^o   Dali, Christine I ^p   de Medeiros, Paula Frassinetti Vasconcelos ^q   Guelbert, Norberto B ^r   Hiwot, Tarekegn G ^t   Jin, Dong Kyu ^u   Jones, Simon A ^v   Lin, Shuan Pei ^w   Maranda, Bruno ^x   Mitchell, John J ^y   Murphy, Elaine Mary ^z   Muschol, Nicole ^aa   Okuyama, Torayuki ^ab   Santra, Saikat ^ac   Villarreal, Martha L Solano ^ad   Steiner, Robert ^ae   Tanpaiboon, Pranoot ^af   Valente, Paula Cristina ^ag   Matos, Santos Baptista Garcia ^ag   Vellodi, Ashok ^o   White, Klane K ^ah   Wijburg, Frits A ^ai   Yang, Ke ^aj   Mealiffe, Matthew ^aj   Haller, Christine ^aj   more..

^a SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^b HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^c CHILDREN S HOSPITAL OAKLAND   (United States)

^d JOHANNES GUTENBERG UNIVERSITY   (Germany)

^e HOSPICES CIVILS DE LYON   (France)

^f HÔPITAL NECKER ENFANTS MALADES   (France)

^g SAN GERARDO HOSPITAL   (Italy)

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

ⁱ NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

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

^k HOSPITAL FOR SICK CHILDREN   (Canada)

^l Qatar Medical Genetics Center   (Qatar)

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

ⁿ CHILDREN'S MEMORIAL HOSPITAL   (United States)

^o GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^p Mental Health Services CPH   (Denmark)

^q FEDERAL UNIVERSITY OF CAMPINA GRANDE   (Brazil)

^r Hospital de Ninos de la Santisima Trinidad   (Argentina)

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

^t SELLY OAK HOSPITAL   (United Kingdom)

^u Samsung Medical Center   (South Korea)

^v MANCHESTER UNIVERSITY NHS FOUNDATION TRUST   (United Kingdom)

^w MACKAY MEMORIAL HOSPITAL   (Taiwan)

^x CENTRE HOSPITALIER UNIVERSITAIRE DE SHERBROOKE   (Canada)

^y MONTREAL CHILDREN S HOSPITAL   (Canada)

^z NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY   (United Kingdom)

^aa UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

^ab NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT   (Japan)

^ac BIRMINGHAM CHILDREN S HOSPITAL   (United Kingdom)

^ad FUNDACIÓN CARDIOINFANTIL INSTITUTO DE CARDIOLOGÍA   (Colombia)

^ae OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^af CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^ag CENTRO HOSPITALAR E UNIVERSITÁRIO DE COIMBRA   (Portugal)

^ah SEATTLE CHILDREN S HOSPITAL   (United States)

^ai EMMA CHILDREN S HOSPITAL   (Netherlands)

^aj BIOMARIN PHARMACEUTICAL INC   (United States)

^ak MATER MISERICORDIAE UNIVERSITY HOSPITAL   (Ireland)

more..

Author keywords

Elosulfase alfa; Enzyme replacement therapy; Lysosomal storage disorder; Morquio A syndrome; Mucopolysaccharidosis IVA; Randomized controlled trial

Indexed keywords

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

ADOLESCENT; ADULT; ANTHROPOMETRY; ARTICLE; BODY GROWTH; BODY HEIGHT; CHILD; CONTROLLED STUDY; CORNEA DISEASE; DAILY LIFE ACTIVITY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; ECHOCARDIOGRAPHY; ENZYME REPLACEMENT; FEMALE; GROWTH RATE; HEALTH ASSESSMENT QUESTIONNAIRE; HEARING TEST; HUMAN; LUNG FUNCTION TEST; LUNG VENTILATION; MAJOR CLINICAL STUDY; MALE; MORQUIO A SYNDROME; MORQUIO SYNDROME; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RADIOGRAPHY; RANDOMIZED CONTROLLED TRIAL; RANK SUM TEST; RESPIRATORY FUNCTION; WALKING; CLINICAL TRIAL; DRUG EFFECTS; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS IV; MULTICENTER STUDY; PATHOPHYSIOLOGY; PRESCHOOL CHILD; QUESTIONNAIRE; TREATMENT OUTCOME; YOUNG ADULT;

IVA;

ACTIVITIES OF DAILY LIVING; ADOLESCENT; ADULT; BODY HEIGHT; CHILD; CHILD, PRESCHOOL; CHONDROITINSULFATASES; DOUBLE-BLIND METHOD; ENZYME REPLACEMENT THERAPY; HUMANS; MAXIMAL VOLUNTARY VENTILATION; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS IV; QUESTIONNAIRES; RESPIRATORY FUNCTION TESTS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84921818034     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2014.08.012     Document Type: Article

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