0000000000597812

AUTHOR

Akemi Tanaka

showing 2 related works from this author

PO-0096 Clinical Effectiveness Of Idursulfase In Boys Aged 0–5 Years With Hunter Syndrome: 3-year Data From The Hunter Outcome Survey

2014

Background and aims Symptoms of Hunter syndrome typically become apparent at 2–4 years of age. Previous analyses have demonstrated improvements in certain clinical measures in young patients receiving idursulfase (Shire); however, data on long-term idursulfase use in these patients remain limited. This analysis used data available in the Hunter Outcome Survey (HOS), a global, observational registry sponsored by Shire, to investigate long-term effectiveness of idursulfase in boys with Hunter syndrome aged 0–5 years. Methods As of January 2014, 260/564 males followed prospectively in HOS had received ≥1 idursulfase infusion (excluding those who had received a bone marrow transplant or were en…

Pediatricsmedicine.medical_specialtyIdursulfaseClinical effectivenessbusiness.industryUrinary systemHunter syndromemedicine.diseaseRheumatologyClinical trialInternal medicinePediatrics Perinatology and Child HealthmedicineObservational studybusinessmedicine.drugLiver sizeArchives of Disease in Childhood
researchProduct

Clinical course of sly syndrome (mucopolysaccharidosis type VII).

2016

WOS: 000377110800007

0301 basic medicineAdultMalePediatricsmedicine.medical_specialtyAdolescentMucopolysaccharidosisSly syndromeHepatosplenomegalyMetabolic disordersMucopolysaccharidosis VIIMedical and Health Sciences03 medical and health sciencesYoung Adult0302 clinical medicineHydrops fetalisSurveys and QuestionnairesmedicineGeneticsHumansMedical history1506Clinical geneticsFamily historyPreschoolChildGenetics (clinical)GlucuronidaseGenetics & Hereditybusiness.industryGenotype-Phenotype CorrelationsMucopolysaccharidosis VIIInfantEnzyme replacement therapyBiological Sciencesmedicine.diseaseLysosomal Storage Diseases030104 developmental biologyPhenotypeClinical genetics Genetics Metabolic disordersChild PreschoolFemalemedicine.symptombusiness030217 neurology & neurosurgeryMPS ; lysosomal storage disease ; β-glucuronidase
researchProduct