Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses-Reference-Cited by-同舟云学术

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Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses

Published:2017-12 Issue: Volume:122 Page:8-16
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Shapiro Elsa G.^ORCID,Escolar Maria L.,Delaney Kathleen A.,Mitchell John J.

Funder

BioMarin Pharmaceutical

Harpur foundation

BioMarin Pharmaceutical Inc.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference94 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Overview of the mucopolysaccharidoses;Muenzer;Rheumatology (Oxford),2011

3. Brain and spinal MR imaging findings in mucopolysaccharidoses: a review;Zafeiriou;AJNR Am. J. Neuroradiol.,2013

4. Why are behaviors of children suffering from various neuronopathic types of mucopolysaccharidoses different?;Węgrzyn;Med. Hypotheses,2010

5. Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations – neurological signs and symptoms;Shapiro;Mol. Genet. Metab.,2017

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1. Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase‐IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II;Journal of Inherited Metabolic Disease;2024-09-09

2. Genotype–phenotype findings in patients with mucopolysaccharidosis II from the Hunter Outcome Survey;Molecular Genetics and Metabolism;2024-09

3. Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment;Orphanet Journal of Rare Diseases;2024-04-12

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5. Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials;BMC Psychiatry;2024-01-04

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