Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome

Molecular Genetics and Metabolism

Volumn 119, Issue 1-2, 2016, Pages 131-143

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

^a SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^b SAN GERARDO HOSPITAL   (Italy)

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

^d HOSPITAL FOR SICK CHILDREN   (Canada)

^e HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^f Diabetes Y Metabolismo ACE   (Colombia)

^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 NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

^m BIOMARIN PHARMACEUTICAL INC   (United States)

ⁿ CHILDREN S HOSPITAL OAKLAND   (United States)

more..

Author keywords

Elosulfase alfa; Endurance; Enzyme replacement therapy; Long term; Morquio A syndrome; Safety

Indexed keywords

CREATININE; DRUG ANTIBODY; ELOSULFASE ALFA; IMMUNOGLOBULIN E; KERATAN SULFATE; PLACEBO; GALNS PROTEIN, HUMAN; N ACETYLGALACTOSAMINE 4 SULFATASE;

ADOLESCENT; ADULT; AGE; ANAPHYLAXIS; ARTICLE; CANE; CHILD; CHRONOPHARMACOLOGY; CLINICAL EVALUATION; COHORT ANALYSIS; CONTROLLED STUDY; CREATININE URINE LEVEL; CRUTCH; DEATH; DOSAGE SCHEDULE COMPARISON; DOUBLE BLIND PROCEDURE; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG FATALITY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENDURANCE; ENZYME REPLACEMENT; FEMALE; FEVER; HEADACHE; HEART FUNCTION TEST; HEMATURIA; HUMAN; HYPERSENSITIVITY; INTENTION TO TREAT ANALYSIS; LONG TERM CARE; LUNG FUNCTION TEST; MAJOR CLINICAL STUDY; MALE; MEDICATION COMPLIANCE; MORQUIO SYNDROME; MULTICENTER STUDY; MULTICENTER STUDY (TOPIC); OPEN STUDY; OPTIMAL DRUG DOSE; ORTHOPEDIC SURGERY; PATIENT COMPLIANCE; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; RECOMMENDED DRUG DOSE; SINGLE DRUG DOSE; SIX MINUTE WALK TEST; THREE MINUTE STAIR CLIMB TEST; TREATMENT DURATION; TREATMENT OUTCOME; URINE LEVEL; VOMITING; WALKER; AGED; CLINICAL TRIAL; DRUG EFFECTS; GENETICS; MIDDLE AGED; PATHOPHYSIOLOGY; PRESCHOOL CHILD; PROCEDURES; URINE; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; CHILD; CHILD, PRESCHOOL; CHONDROITINSULFATASES; DOUBLE-BLIND METHOD; ENZYME REPLACEMENT THERAPY; FEMALE; HUMANS; KERATAN SULFATE; MALE; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS IV; PHYSICAL ENDURANCE; YOUNG ADULT;

EID: 84991011549     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2016.05.018     Document Type: Article

References (12)

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