Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
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Title
Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
Authors
Keywords
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Journal
Orphanet Journal of Rare Diseases
Volume 16, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2021-12-04
DOI
10.1186/s13023-021-02118-3
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- (2018) Maria Mattera et al. Health and Quality of Life Outcomes
- Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure
- (2017) Johanna H. van der Lee et al. MOLECULAR GENETICS AND METABOLISM
- Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature
- (2017) Darren Janzen et al. MOLECULAR GENETICS AND METABOLISM
- Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses
- (2017) Elsa G. Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- Practical management of behavioral problems in mucopolysaccharidoses disorders
- (2017) Maria L. Escolar et al. MOLECULAR GENETICS AND METABOLISM
- Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
- (2017) Joseph Muenzer et al. Orphanet Journal of Rare Diseases
- Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future
- (2017) David Whiteman et al. Drug Design Development and Therapy
- Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions
- (2016) Elsa Shapiro et al. MOLECULAR GENETICS AND METABOLISM
- A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
- (2015) Joseph Muenzer et al. GENETICS IN MEDICINE
- The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: item reduction and further validation
- (2014) Ingela Wiklund et al. QUALITY OF LIFE RESEARCH
- MPS II: Adaptive Behavior of Patients and Impact on the Family System
- (2013) Mary Needham et al. Journal of Genetic Counseling
- The impact of Hunter syndrome (mucopolysaccharidosis type II) on health-related quality of life
- (2013) Mireia Raluy-Callado et al. Orphanet Journal of Rare Diseases
- CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder
- (2012) Pericles Calias et al. PLoS One
- The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: evaluation of measurement properties
- (2012) Ingela Wiklund et al. QUALITY OF LIFE RESEARCH
- Natural Progression of Neurological Disease in Mucopolysaccharidosis Type II
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- (2010) Joseph Muenzer et al. GENETICS IN MEDICINE
- Initial report from the Hunter Outcome Survey
- (2009) J Edmond Wraith et al. GENETICS IN MEDICINE
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