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Therapy development for the mucopolysaccharidoses : updated consensus recommendations for neuropsychological endpoints
dc.contributor.author | Van der Lee, Johanna Hanneke | pt_BR |
dc.contributor.author | Morton, Jonathan | pt_BR |
dc.contributor.author | Adams, Heather R. | pt_BR |
dc.contributor.author | Clarke, Lorne A. | pt_BR |
dc.contributor.author | Eisengart, Julie B. | pt_BR |
dc.contributor.author | Escolar, Maria Luisa | pt_BR |
dc.contributor.author | Giugliani, Roberto | pt_BR |
dc.contributor.author | Harmatz, Paul | pt_BR |
dc.contributor.author | Hogan, Melissa | pt_BR |
dc.contributor.author | Kearney, Shauna | pt_BR |
dc.contributor.author | Muenzer, Joseph | pt_BR |
dc.contributor.author | Muschol, Nicole | pt_BR |
dc.contributor.author | Rust, Stewart | pt_BR |
dc.contributor.author | Saville, Benjamin R. | pt_BR |
dc.contributor.author | Semrud-Clikeman, Margaret T. | pt_BR |
dc.contributor.author | Wang, Raymond Y. | pt_BR |
dc.contributor.author | Shapiro, Elsa G. | pt_BR |
dc.date.accessioned | 2021-05-13T04:27:42Z | pt_BR |
dc.date.issued | 2020 | pt_BR |
dc.identifier.issn | 1096-7206 | pt_BR |
dc.identifier.uri | http://hdl.handle.net/10183/220906 | pt_BR |
dc.description.abstract | Neurological dysfunction represents a significant clinical component of many of the mucopolysaccharidoses (also known as MPS disorders). The accurate and consistent assessment of neuropsychological function is essential to gain a greater understanding of the precise natural history of these conditions and to design effective clinical trials to evaluate the impact of therapies on the brain. In 2017, an International MPS Consensus Panel published recommendations for best practice in the design and conduct of clinical studies investigating the effects of therapies on cognitive function and adaptive behavior in patients with neuronopathic mucopolysaccharidoses. Based on an International MPS Consensus Conference held in February 2020, this article provides updated consensus recommendations and expands the objectives to include approaches for assessing behavioral and social-emotional state, caregiver burden and quality of life in patients with all mucopolysaccharidoses. | en |
dc.format.mimetype | application/pdf | pt_BR |
dc.language.iso | eng | pt_BR |
dc.relation.ispartof | Molecular genetics and metabolism. Amsterdam. Vol. 131, no. 1-2 (2020), p. 181-196 | pt_BR |
dc.rights | Open Access | en |
dc.subject | Mucopolissacaridoses | pt_BR |
dc.subject | Mucopolysaccharidoses | en |
dc.subject | Cognição | pt_BR |
dc.subject | Cognitive | en |
dc.subject | Behavior | en |
dc.subject | Consenso | pt_BR |
dc.subject | Protocolos clínicos | pt_BR |
dc.subject | Social-emotional | en |
dc.subject | Comportamento | pt_BR |
dc.subject | Clinical trial | en |
dc.subject | Estudo clínico | pt_BR |
dc.subject | Protocol | en |
dc.title | Therapy development for the mucopolysaccharidoses : updated consensus recommendations for neuropsychological endpoints | pt_BR |
dc.type | Artigo de periódico | pt_BR |
dc.identifier.nrb | 001121833 | pt_BR |
dc.type.origin | Estrangeiro | pt_BR |
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