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Enzyme replacement therapy in Hurler syndrome after failure of hematopoietic transplant

Leonor Arranz1, Luis Aldamiz-Echevarria2

  • 1Pediatrics Department, Donostia Universitary Hospital, San Sebastian, Spain; Pediatrics Department, Faculty of Medicine, Basque Country University (UPV/EHU), San Sebastian, Spain.

Molecular Genetics and Metabolism Reports
|March 4, 2016
PubMed

Keywords:
6MWT, 6-minute walk testAnti-laronidase antibodiesERT, Enzyme replacement therapyEnzyme replacement therapyFEV0.5, forced expiratory volume in 0.5 sFVC, forced vital capacityGAGs, glycosaminoglycansHSCT, hematopoietic stem cell transplantationHematopoietic transplantHurler syndromeIV, intravenousLaronidaseMMEF, maximum midexpiratory flowMPS-I, mucopolysaccharidosis type IMRI, magnetic Resonance ImagingMSCA, McCarthy Scales of Children's AbilitiesMucopolysaccharosidosis type IORL, otorhinolaryngologyOSAHS, Obstructive Sleep Apnea–Hypopnea SyndromeTMT (A), Trail Making Test Part AWISC-IV, Wechsler Intelligence Scale for Children — IVX-ray, radiograph

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