Mucopolysaccharidosis VI: The Italian experience

European Journal of Pediatrics

Volumn 168, Issue 10, 2009, Pages 1203-1206

  Scarpa, Maurizio ^a   Barone, Rita ^b   Fiumara, Agata ^b   Astarita, Luca ^c   Parenti, Giancarlo ^c,d   Rampazzo, Angelica ^a   Sala, Stefania ^e   Sorge, Giovanni ^b   Parini, Rossella ^e  

^a UNIVERSITY OF PADOVA   (Italy)

^b UNIVERSITY OF CATANIA   (Italy)

^c UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^d TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

^e SAN GERARDO HOSPITAL   (Italy)

Author keywords

Enzyme replacement therapy; MPS; Mucopolysaccharidoses; Mucopolysaccharidosis VI; Naglazyme

Indexed keywords

GALSULFASE;

ADOLESCENT; APNEA; ARTICLE; BODY GROWTH; BODY WEIGHT; BONE DISEASE; CARPAL TUNNEL SYNDROME; CATARACT; CHILD; CLINICAL ARTICLE; CONTRACTURE; DISEASE COURSE; ENZYME REPLACEMENT; EYE DISEASE; FACIES; FEMALE; HEARING IMPAIRMENT; HEPATOSPLENOMEGALY; HIP MALFORMATION; HUMAN; HYPOSPADIAS; ITALY; JOINT MOBILITY; JOINT STIFFNESS; KYPHOSIS; MACROCEPHALY; MALE; MAROTEAUX LAMY SYNDROME; NEUROLOGIC DISEASE; PRESCHOOL CHILD; PRIORITY JOURNAL; RESPIRATORY TRACT DISEASE; RIB MALFORMATION; SCHOOL CHILD; SCOLIOSIS; SHORT STATURE; SKELETON MALFORMATION; SPINAL CORD COMPRESSION; URINARY TRACT INFECTION; VALGUS KNEE; VALVULAR HEART DISEASE; VISUAL IMPAIRMENT;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; FEMALE; HUMANS; ITALY; MALE; MUCOPOLYSACCHARIDOSIS VI; TREATMENT OUTCOME;

EID: 69049101187     PISSN: 03406199     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00431-008-0910-z     Document Type: Article

References (8)

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6. P Harmatz R Giugliani IVD Schwartz, et al. 2008 Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase Mol Genet Metab 94 469 475
7. P Harmatz D Ketteridge R Giugliani, et al. 2005 Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase Pediatrics 115 e681 e689
8. P Harmatz CB Whitley L Waber, et al. 2004 Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) J Pediatr 144 574 580