Please use this identifier to cite or link to this item: http://hdl.handle.net/1942/29865
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dc.contributor.authorMayhew, Anna G.-
dc.contributor.authorCoratti, Giorgia-
dc.contributor.authorMazzone, Elena Stacy-
dc.contributor.authorKLINGELS, Katrijn-
dc.contributor.authorJames, Meredith-
dc.contributor.authorPane, Marika-
dc.contributor.authorStraub, Volker-
dc.contributor.authorGoemans, Natalie-
dc.contributor.authorMercuri, Eugenio-
dc.contributor.authorRicotti, Valeria-
dc.contributor.authorMuntoni, Francesco-
dc.contributor.authorRidout, Deborah-
dc.contributor.authorSelby, Victoria-
dc.date.accessioned2019-10-28T13:24:07Z-
dc.date.available2019-10-28T13:24:07Z-
dc.date.issued2019-
dc.identifier.citationDEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY, 62(5), pp. 633-639-
dc.identifier.issn0012-1622-
dc.identifier.urihttp://hdl.handle.net/1942/29865-
dc.description.abstractAIM To report the differences between Performance of Upper Limb (PUL) versions 1.2 and 2.0, compare the measurement ability of the two versions, and compare their longitudinal performance in Duchenne muscular dystrophy. METHOD Rasch analysis was performed on the dual data from three centres to confirm whether the two scales measure the same construct. Change scores in natural history for the different domains were compared for the two versions. RESULTS Rasch analysis demonstrated that both versions measure the same construct and that the PUL 2.0 was a better fit to the construct of motor performance and better able to detect change at 12 months in all levels of ability than the PUL 1.2. This was also true when change scores were reviewed over 2 years. INTERPRETATION Our results confirm that the PUL 1.2 and 2.0 versions detect change in all domains over 2 years. They also demonstrate that simplifying the original scoring of the PUL 1.2 for the revised PUL 2.0 maintains the validity of the construct and enhances the scale measurement qualities.-
dc.description.sponsorshipThe members of the PUL Working Group are as follows: Valeria Ricotti, Francesco Muntoni, Deborah Ridout, and Victoria Selby. VR is currently employed by Solid Biosciences. FM is a member of the Rare Disease Scientific Advisory Group for Pfizer and has participated in scientific advisory board meetings for PTC, Sarepta, Summit, and Wave Therapeutics. UCL and Great Ormond Street Hospital are recipients of grants from Pfizer, Sarepta, Italfarmaco, Wave Therapeutics, and Summit regarding clinical trials (Muntoni PI). We acknowledge the contribution of the North Star Network (https://www.northstardmd.com), in particular Newcastle. The financial support of MDUK for the North Star Project (AM and FM) and of the MRC Translational Research Centre to UCL and Newcastle (MR/K501074/1) is gratefully acknowledged. This research was supported by the NIHR GOSH BRC and the NIHR Newcastle BRC. The views expressed are those of the authors and not necessarily those of the NHS, the NIHR, or the Department of Health. The authors' conflicts of interest are available in Appendix S2 (online supporting information).-
dc.language.isoen-
dc.publisherWILEY-
dc.rights2019 Mac Keith Press-
dc.titlePerformance of Upper Limb module for Duchenne muscular dystrophy-
dc.typeJournal Contribution-
dc.identifier.epage639-
dc.identifier.issue5-
dc.identifier.spage633-
dc.identifier.volume62-
local.format.pages7-
local.bibliographicCitation.jcatA1-
dc.description.notes[Mayhew, Anna G.; James, Meredith; Straub, Volker] Newcastle Univ, John Walton Muscular Dystrophy Res Ctr, Inst Genet Med, Newcastle Upon Tyne, Tyne & Wear, England. [Mayhew, Anna G.; James, Meredith; Straub, Volker] Newcastle Hosp NHS Fdn Trust, Cent Pkwy, Newcastle Upon Tyne NE1 3BZ, Tyne & Wear, England. [Coratti, Giorgia; Mazzone, Elena Stacy; Pane, Marika; Mercuri, Eugenio] Univ Cattolica Sacro Cuore, IRCCS Fdn Policlin Gemelli, Child Neurol Unit, Rome, Italy. [Coratti, Giorgia; Mazzone, Elena Stacy; Pane, Marika; Mercuri, Eugenio] Univ Cattolica Sacro Cuore, IRCCS Fdn Policlin Gemelli, Ctr Nemo, Rome, Italy. [Klingels, Katrijn] Univ Leuven, Dept Rehabil Sci, KU Leuven, Heverlee, Belgium. [Klingels, Katrijn] Hasselt Univ, Fac Rehabil Sci, REVAL, Hasselt, Belgium. [Goemans, Natalie] Univ Hosp Leuven, Child Neurol, Leuven, Belgium.-
local.publisher.placeHOBOKEN-
local.type.refereedRefereed-
local.type.specifiedArticle-
dc.identifier.doi10.1111/dmcn.14361-
dc.identifier.isi000487491400001-
item.validationecoom 2020-
item.accessRightsOpen Access-
item.fullcitationMayhew, Anna G.; Coratti, Giorgia; Mazzone, Elena Stacy; KLINGELS, Katrijn; James, Meredith; Pane, Marika; Straub, Volker; Goemans, Natalie; Mercuri, Eugenio; Ricotti, Valeria; Muntoni, Francesco; Ridout, Deborah & Selby, Victoria (2019) Performance of Upper Limb module for Duchenne muscular dystrophy. In: DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY, 62(5), pp. 633-639.-
item.fulltextWith Fulltext-
item.contributorMayhew, Anna G.-
item.contributorCoratti, Giorgia-
item.contributorMazzone, Elena Stacy-
item.contributorKLINGELS, Katrijn-
item.contributorJames, Meredith-
item.contributorPane, Marika-
item.contributorStraub, Volker-
item.contributorGoemans, Natalie-
item.contributorMercuri, Eugenio-
item.contributorRicotti, Valeria-
item.contributorMuntoni, Francesco-
item.contributorRidout, Deborah-
item.contributorSelby, Victoria-
crisitem.journal.issn0012-1622-
crisitem.journal.eissn1469-8749-
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