Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)-10-year follow-up of patients who previously participated in an MPS VI survey study

American Journal of Medical Genetics, Part A

Volumn 164, Issue 8, 2014, Pages 1953-1964

  Giugliani, Roberto ^a   Lampe, Christina ^b   Guffon, Nathalie ^c   Ketteridge, David ^d   Leão Teles, Elisa ^e   Wraith, James E ^f   Jones, Simon A ^f   Piscia Nichols, Cheri ^g   Lin, Ping ^g   Quartel, Adrian ^g   Harmatz, Paul ^h  

^a HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^b UNIVERSITY MEDICAL CENTER MAINZ   (Germany)

^c HOSPICES CIVILS DE LYON   (France)

^d WOMEN S AND CHILDREN S HOSPITAL   (Australia)

^e Universidade do Porto   (Portugal)

^f UNIVERSITY OF MANCHESTER   (United Kingdom)

^g BIOMARIN PHARMACEUTICAL INC   (United States)

^h CHILDREN S HOSPITAL OAKLAND   (United States)

Author keywords

Enzyme replacement therapy; Exercise tolerance; Follow up studies; Maroteaux Lamy syndrome; Mucopolysaccharidosis VI; Multicenter study publication type ; N acetylgalactosamine 4 sulfatase; Respiratory function tests; Survival rate

Indexed keywords

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

6 MINUTE WALK TEST; ADOLESCENT; ADULT; ANTHROPOMETRY; ARTICLE; BODY HEIGHT; CARDIOVASCULAR PARAMETERS; CHILD; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CROSS-SECTIONAL STUDY; DISEASE COURSE; ENDURANCE; ENZYME REPLACEMENT; FEMALE; FORCED EXPIRATORY VOLUME; FORCED VITAL CAPACITY; FUNCTION TEST; HEART FUNCTION; HEART VENTRICLE HYPERTROPHY; HEART VENTRICLE SIZE; HUMAN; LEFT VENTRICULAR SHORTENING FRACTION; LONG TERM CARE; LONG TERM SURVIVAL; LUNG FUNCTION; LUNG FUNCTION TEST; MAJOR CLINICAL STUDY; MALE; MAROTEAUX LAMY SYNDROME; MEDICAL HISTORY; MORTALITY; MULTICENTER STUDY (TOPIC); OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUALITY OF LIFE; SINUS TACHYCARDIA; TREATMENT DURATION; URINE LEVEL; CLINICAL TRIAL; EXERCISE TEST; FOLLOW UP; HEART FUNCTION TEST; MORPHOMETRICS; MUCOPOLYSACCHARIDOSIS VI; MULTICENTER STUDY; PRESCHOOL CHILD; URINE; YOUNG ADULT;

ADOLESCENT; BODY WEIGHTS AND MEASURES; CHILD; CHILD, PRESCHOOL; CROSS-SECTIONAL STUDIES; ENZYME REPLACEMENT THERAPY; EXERCISE TEST; FEMALE; FOLLOW-UP STUDIES; HEART FUNCTION TESTS; HUMANS; MALE; MUCOPOLYSACCHARIDOSIS VI; N-ACETYLGALACTOSAMINE-4-SULFATASE; QUALITY OF LIFE; RECOMBINANT PROTEINS; RESPIRATORY FUNCTION TESTS; YOUNG ADULT;

EID: 84904416919     PISSN: 15524825     EISSN: 15524833     Source Type: Journal    
DOI: 10.1002/ajmg.a.36584     Document Type: Article

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