Enzyme replacement therapy for mucopolysaccharidosis VI: Long-term cardiac effects of galsulfase (Naglazyme®) therapy

Journal of Inherited Metabolic Disease

Volumn 36, Issue 2, 2013, Pages 385-394

  Braunlin, E ^a   Rosenfeld, H ^b   Kampmann, C ^c   Johnson, J ^b   Beck, M ^c   Giugliani, R ^d,e,f   Guffon, N ^g   Ketteridge, D ^h   Sa Miranda C M ⁱ   Scarpa, M ^j   Schwartz, I V ^d,e   Leao Teles E ^k   Wraith, J E ^l   Barrios, P ^d,e   Dias Da Silva, E ^k   Kurio, G ^b   Richardson, M ^h   Gildengorin, G ^b   Hopwood, J J ^h   Imperiale, M ^m   Schatz, A ^m   Decker, C ^m   Harmatz, P ^b   more..

^a UNIVERSITY OF MINNESOTA   (United States)

^b CHILDREN S HOSPITAL OAKLAND   (United States)

^c UNIVERSITY MEDICAL CENTER MAINZ   (Germany)

^d FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^e HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^f Instituto Nacional de Genética Médica Populacional INAGEMP   (Brazil)

^g HOSPICES CIVILS DE LYON   (France)

^h WOMEN S AND CHILDREN S HOSPITAL   (Australia)

ⁱ UNIVERSITY OF PORTO   (Portugal)

^j UNIVERSITY OF PADOVA   (Italy)

^k HOSPITAL DE SÃO JOÃO   (Portugal)

^l ST MARY S HOSPITAL   (United Kingdom)

^m BIOMARIN PHARMACEUTICAL INC   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

GALSULFASE; GLYCOSAMINOGLYCAN; PLACEBO; N ACETYLGALACTOSAMINE 4 SULFATASE; RECOMBINANT PROTEIN;

ADOLESCENT; ADULT; AORTA VALVE REGURGITATION; ARTICLE; CHILD; ECHOCARDIOGRAPHY; ENZYME REPLACEMENT; FEMALE; HEART LEFT VENTRICLE FUNCTION; HEART LEFT VENTRICLE HYPERTROPHY; HEART VALVE STENOSIS; HEART VENTRICLE SEPTUM; HUMAN; LONG TERM CARE; LOW DRUG DOSE; MAJOR CLINICAL STUDY; MALE; MAROTEAUX LAMY SYNDROME; MITRAL VALVE REGURGITATION; MITRAL VALVE REPLACEMENT; MITRAL VALVE STENOSIS; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRESCHOOL CHILD; RANDOMIZED CONTROLLED TRIAL (TOPIC); SCHOOL CHILD; CHEMICALLY INDUCED; CLINICAL TRIAL (TOPIC); DRUG EFFECTS; HEART VALVE; MUCOPOLYSACCHARIDOSIS VI; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADULT; CHILD; CLINICAL TRIALS AS TOPIC; ENZYME REPLACEMENT THERAPY; FEMALE; HEART VALVES; HUMANS; HYPERTROPHY, LEFT VENTRICULAR; MALE; MUCOPOLYSACCHARIDOSIS VI; N-ACETYLGALACTOSAMINE-4-SULFATASE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECOMBINANT PROTEINS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84879684477     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-012-9481-2     Document Type: Article

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