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The Neurobehavioral Phenotype in Mucopolysaccharidosis Type IIIB: an Exploratory Study.A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA.Association of somatic burden of disease with age and neuropsychological measures in attenuated mucopolysaccharidosis types I, II and VI.A longitudinal study of emotional adjustment, quality of life and adaptive function in attenuated MPS IINeurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions.Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature.Observing the advanced disease course in mucopolysaccharidosis, type IIIA; a case series.Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses.Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations - Neurological signs and symptoms.Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trialAnalysis of the caregiver burden associated with Sanfilippo syndrome type B: panel recommendations based on qualitative and quantitative data
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description
investigador
@es
researcher
@en
wetenschapper
@nl
name
Elsa G Shapiro
@en
Elsa G Shapiro
@nl
type
label
Elsa G Shapiro
@en
Elsa G Shapiro
@nl
prefLabel
Elsa G Shapiro
@en
Elsa G Shapiro
@nl
P31
P496
0000-0003-0276-0276