Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients

Molecular Genetics and Metabolism Reports

Volumn 3, Issue , 2015, Pages 65-74

  Parini, Rossella ^a   Rigoldi, Miriam ^a   Tedesco, Lucia ^a   Boffi, Lucia ^a   Brambilla, Alessandra ^a   Bertoletti, Sara ^a   Boncimino, Agata ^a,d   Del Longo, Alessandra ^b   De Lorenzo, Paola ^c   Gaini, Renato ^a   Gallone, Denise ^a   Gasperini, Serena ^a   Giussani, Carlo ^a   Grimaldi, Marco ^a,e   Grioni, Daniele ^a   Meregalli, Pamela ^a   Messinesi, Grazia ^a   Nichelli, Francesca ^a   Romagnoli, Marco ^a   Russo, Pierluigi ^a,f   Sganzerla, Erik ^a   Valsecchi, Grazia ^c   Biondi, Andrea ^a   more..

^a SAN GERARDO HOSPITAL   (Italy)

^b OSPEDALE NIGUARDA CA GRANDA   (Italy)

^c UNIVERSITY OF MILANO BICOCCA   (Italy)

^d Dept of Pediatrics Azienda Ospedaliera San Carlo   (Italy)

^e Dept of Pediatric Ophthalmology Niguarda Hospital ^*  (Italy)

^f Dept of Cardiology Azienda Ospedaliera ASL Foggia Ospedale G Tatarella Cerignola   (Italy)

Author keywords

Enzymatic replacement therapy; ERT; Hunter disease; Hunter syndrome; Idursulfase; MPS II; Mucopolysaccharidosis type II

Indexed keywords

IDURONATE 2 SULFATASE;

ADULT; ANTHROPOMETRY; ARTICLE; CARPAL TUNNEL SYNDROME; CLINICAL ARTICLE; CRANIOFACIAL SYNOSTOSIS; DRUG SAFETY; DRUG WITHDRAWAL; ENZYME REPLACEMENT; FOLLOW UP; FOOT MALFORMATION; FORCED VITAL CAPACITY; HEART LEFT VENTRICLE MASS; HEPATOMEGALY; HIP DYSPLASIA; HUMAN; HUNTER SYNDROME; HYPEROSTOSIS; KYPHOSIS; LIVER SIZE; LORDOSIS; MALE; OBSERVATIONAL STUDY; PHASE 3 CLINICAL TRIAL; PHENOTYPE; PROSPECTIVE STUDY; PRURITUS; QUALITY OF LIFE; RANGE OF MOTION; RESPIRATORY FUNCTION; SCOLIOSIS; SPLEEN SIZE; SPLENOMEGALY; TREATMENT OUTCOME; URTICARIA; VALGUS KNEE; VERTEBRAL CANAL STENOSIS;

EID: 84928493219     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2015.03.011     Document Type: Article

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