Matches in SemOpenAlex for { <https://semopenalex.org/work/W2612749062> ?p ?o ?g. }
- W2612749062 endingPage "69" @default.
- W2612749062 startingPage "57" @default.
- W2612749062 abstract "Sensitive, reliable measurement instruments are critical for the evaluation of disease progression and new treatments that affect the brain in the mucopolysaccharidoses (MPS). MPS I, II, and III have early onset clinical phenotypes that affect the brain during development and result in devastating cognitive decline and ultimately death without treatment. Comparisons of outcomes are hindered by diverse protocols and approaches to assessment including applicability to international trials necessary in rare diseases. We review both cognitive and adaptive measures with the goal of providing evidence to a Delphi panel to come to a consensus about recommendations for clinical trials for various age groups. The results of the consensus panel are reported in an accompanying article. The following data were gathered (from internet resources and from test manuals) for each measure and summarized in the discussion: reliability, validity, date and adequacy of normative data, applicability of the measure's metrics, cross cultural validity including translations and adaptations, feasibility in the MPS population, familiarity to sites, sensitivity to change, and interpretability. If, resulting from this consensus, standard protocols are used for both natural history and treatment studies, patients, their families, and health care providers will benefit from the ability to compare study outcomes." @default.
- W2612749062 created "2017-05-19" @default.
- W2612749062 creator A5000102952 @default.
- W2612749062 creator A5054062079 @default.
- W2612749062 creator A5064200493 @default.
- W2612749062 date "2017-06-01" @default.
- W2612749062 modified "2023-09-26" @default.
- W2612749062 title "Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature" @default.
- W2612749062 cites W1493161648 @default.
- W2612749062 cites W1541729841 @default.
- W2612749062 cites W1563374141 @default.
- W2612749062 cites W156410711 @default.
- W2612749062 cites W1571262084 @default.
- W2612749062 cites W1646942865 @default.
- W2612749062 cites W1832822495 @default.
- W2612749062 cites W1894914874 @default.
- W2612749062 cites W1943855526 @default.
- W2612749062 cites W1968258454 @default.
- W2612749062 cites W1971215307 @default.
- W2612749062 cites W1971427674 @default.
- W2612749062 cites W1971977235 @default.
- W2612749062 cites W1972413162 @default.
- W2612749062 cites W1973635706 @default.
- W2612749062 cites W1981969305 @default.
- W2612749062 cites W1985193566 @default.
- W2612749062 cites W1991511506 @default.
- W2612749062 cites W1991633412 @default.
- W2612749062 cites W1991745146 @default.
- W2612749062 cites W1993027304 @default.
- W2612749062 cites W1997831257 @default.
- W2612749062 cites W1999779579 @default.
- W2612749062 cites W2001556991 @default.
- W2612749062 cites W2001598331 @default.
- W2612749062 cites W2006202967 @default.
- W2612749062 cites W2009046292 @default.
- W2612749062 cites W2012775152 @default.
- W2612749062 cites W2014189616 @default.
- W2612749062 cites W2016279051 @default.
- W2612749062 cites W2020409398 @default.
- W2612749062 cites W2021041107 @default.
- W2612749062 cites W2021349780 @default.
- W2612749062 cites W202348765 @default.
- W2612749062 cites W2023755779 @default.
- W2612749062 cites W2026477204 @default.
- W2612749062 cites W2028570395 @default.
- W2612749062 cites W2030481688 @default.
- W2612749062 cites W2034981978 @default.
- W2612749062 cites W2037692931 @default.
- W2612749062 cites W2037812198 @default.
- W2612749062 cites W2049400700 @default.
- W2612749062 cites W2050953889 @default.
- W2612749062 cites W2051525813 @default.
- W2612749062 cites W2052510697 @default.
- W2612749062 cites W2060496255 @default.
- W2612749062 cites W2064410183 @default.
- W2612749062 cites W2066146740 @default.
- W2612749062 cites W2068792891 @default.
- W2612749062 cites W2071812157 @default.
- W2612749062 cites W2072711133 @default.
- W2612749062 cites W2072725139 @default.
- W2612749062 cites W2076191063 @default.
- W2612749062 cites W2076817651 @default.
- W2612749062 cites W2080568560 @default.
- W2612749062 cites W2082224609 @default.
- W2612749062 cites W2082548895 @default.
- W2612749062 cites W2085884984 @default.
- W2612749062 cites W2087050432 @default.
- W2612749062 cites W2089832931 @default.
- W2612749062 cites W2100217935 @default.
- W2612749062 cites W2101558777 @default.
- W2612749062 cites W2106564695 @default.
- W2612749062 cites W2118780982 @default.
- W2612749062 cites W2121882313 @default.
- W2612749062 cites W2123587476 @default.
- W2612749062 cites W2131677792 @default.
- W2612749062 cites W2134704141 @default.
- W2612749062 cites W2135684115 @default.
- W2612749062 cites W2136255603 @default.
- W2612749062 cites W2137593614 @default.
- W2612749062 cites W2143721978 @default.
- W2612749062 cites W2145402228 @default.
- W2612749062 cites W2149544529 @default.
- W2612749062 cites W2151463239 @default.
- W2612749062 cites W2152901044 @default.
- W2612749062 cites W2165282314 @default.
- W2612749062 cites W2165541265 @default.
- W2612749062 cites W2168218655 @default.
- W2612749062 cites W2168453491 @default.
- W2612749062 cites W2171050058 @default.
- W2612749062 cites W2171762906 @default.
- W2612749062 cites W2216662624 @default.
- W2612749062 cites W2267901974 @default.
- W2612749062 cites W2288903345 @default.
- W2612749062 cites W2296003285 @default.
- W2612749062 cites W2303018029 @default.
- W2612749062 cites W2314320566 @default.
- W2612749062 cites W2314508473 @default.
- W2612749062 cites W2322558233 @default.