0000000000132895

AUTHOR

David A.h. Whiteman

showing 5 related works from this author

Correction: Corrigendum: Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

2013

CORRIGENDUM: Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

Veterinary medicinePediatricsmedicine.medical_specialtybusiness.industryIdursulfaseExtension studyHunter syndromesocial sciencesmedicine.diseasehumanitiesbehavior and behavior mechanismsmedicinepopulation characteristicsbusinessgeographic locationsGenetics (clinical)medicine.drugGenetics in Medicine
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Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B

2017

To evaluate the natural course of disease progression in patients with Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB), identify potential end points for future therapy trials, and characterize biomarkers related to the disease.A prospective, multicenter study was conducted. Baseline, 6-month, and 12-month assessments included neurodevelopmental status (Bayley Scales of Infant Development, Third edition), adaptive status (Vineland Adaptive Behavior Scales, Second Edition), volumetric brain magnetic resonance imaging, cerebrospinal fluid heparan sulfate, and urine glycosaminoglycan (GAG) measurements.Nineteen patients aged 1.6-31.7 years were enrolled. Over 12 months, cognition,…

AdultMale0301 basic medicinePediatricsmedicine.medical_specialtyAdolescentDiseaseBayley Scales of Infant DevelopmentMucopolysaccharidosis IIIYoung Adult03 medical and health sciences0302 clinical medicineMucopolysaccharidosis IIImedicineHumansLongitudinal StudiesProspective StudiesYoung adultChildProspective cohort studyCerebrospinal FluidGlycosaminoglycansSanfilippo syndromemedicine.diagnostic_testbusiness.industryBrainInfantMagnetic resonance imagingmedicine.diseaseMagnetic Resonance ImagingVineland Adaptive Behavior Scale030104 developmental biologyNeurodevelopmental DisordersChild PreschoolPediatrics Perinatology and Child HealthDisease ProgressionFemaleHeparitin SulfatebusinessBiomarkers030217 neurology & neurosurgeryThe Journal of Pediatrics
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Insights into the management of patients with MPS II: Key findings following 10years of the Hunter Outcome Survey (HOS)

2016

medicine.medical_specialtyEndocrinologybusiness.industryEndocrinology Diabetes and MetabolismGeneticsKey (cryptography)MedicinebusinessIntensive care medicineMolecular BiologyBiochemistryOutcome (game theory)Molecular Genetics and Metabolism
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Enzyme replacement therapy in MPS II patients over 20years of age or older at treatment initiation: An international case series from the Hunter Outc…

2013

Series (stratigraphy)medicine.medical_specialtybusiness.industryEndocrinology Diabetes and MetabolismEnzyme replacement therapyBiochemistryOutcome (game theory)SurgeryEndocrinologyInternal medicineGeneticsMedicinebusinessMolecular BiologyMolecular Genetics and Metabolism
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Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.

2011

Purpose: This study evaluated the safety and effectiveness of long-term enzyme replacement therapy with idursulfase (recombinant human iduronate-2-sulfatase) in patients with Hunter syndrome. Methods: All 94 patients who completed a 53-week double-blinded study of idursulfase enrolled in this open-labeled extension study and received intravenous idursulfase at a dose of 0.5 mg/kg weekly for 2 years, and clinical outcomes and safety were assessed. Results: No change in percent predicted forced vital capacity was seen, but absolute forced vital capacity demonstrated sustained improvement and was increased 25.1% at the end of the study. Statistically significant increases in 6-minute walking t…

medicine.medical_specialtyVital capacityAdolescentIdursulfaseIduronate SulfatasePulmonary function testingInternal medicineMedicineHumansEnzyme Replacement TherapyMucopolysaccharidosis type IIAdverse effectChildInfusions IntravenousGenetics (clinical)GlycosaminoglycansMucopolysaccharidosis IIbusiness.industryPercent Predicted Forced Vital CapacityHunter syndromeEnzyme replacement therapyOrgan Sizemedicine.diseaseSurgeryTreatment OutcomeLiverChild PreschoolbusinessSpleenmedicine.drugGenetics in medicine : official journal of the American College of Medical Genetics
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