TheadventoftherapeuticapproachesforDuchennemusculardystrophy(DMD)has highlightedtheneedtoidentifyreliableoutcomemeasuresforyoungboyswithDMD.The aimofthisstudywastodeveloparevisedversion oftheNorth StarAmbulatoryAssessment (NSAA)suitableforboysbetweentheageof3and5yearsbyidentifying ageappropriate itemsandrevisingthescoringsystemaccordingly.Usingthescalein171controlsbetween theageof2.9and4.8years,weidentifieditemsthatwereappropriateatdifferent age points.Anitemwasdefinedasageappropriateifitwascompleted,achievingafullscore, byatleast85%ofthetypicallydevelopingboysatthatage.At3years(±3months)there wereonly8itemsthatwereageappropriate,at3yearsand6monthstherewere13items whilebytheageof4yearsall17itemswereappropriate.Arevisedversionofthescalewas developedwithitemsorderedaccordingtotheagewhentheycouldbereliablyperformed. TheapplicationoftherevisedversionofthescaletodatacollectedinyoungDMDboys showedthatveryfewoftheDMDboyswereabletocompletewithafullscorealltheage appropriateitems.Inconclusion,ourstudysuggeststhatarevisedversionoftheNSAAcan beusedinboysfromtheageof3yearstoobtaininformationonhowyoungDMDboys acquirenewabilitiesandhowthiscorrelateswiththeirpeers.
Revised north star ambulatory assessment for young boys with Duchenne muscular dystrophy
MESSINA, Sonia;Vita, Gian Luca;
2016-01-01
Abstract
TheadventoftherapeuticapproachesforDuchennemusculardystrophy(DMD)has highlightedtheneedtoidentifyreliableoutcomemeasuresforyoungboyswithDMD.The aimofthisstudywastodeveloparevisedversion oftheNorth StarAmbulatoryAssessment (NSAA)suitableforboysbetweentheageof3and5yearsbyidentifying ageappropriate itemsandrevisingthescoringsystemaccordingly.Usingthescalein171controlsbetween theageof2.9and4.8years,weidentifieditemsthatwereappropriateatdifferent age points.Anitemwasdefinedasageappropriateifitwascompleted,achievingafullscore, byatleast85%ofthetypicallydevelopingboysatthatage.At3years(±3months)there wereonly8itemsthatwereageappropriate,at3yearsand6monthstherewere13items whilebytheageof4yearsall17itemswereappropriate.Arevisedversionofthescalewas developedwithitemsorderedaccordingtotheagewhentheycouldbereliablyperformed. TheapplicationoftherevisedversionofthescaletodatacollectedinyoungDMDboys showedthatveryfewoftheDMDboyswereabletocompletewithafullscorealltheage appropriateitems.Inconclusion,ourstudysuggeststhatarevisedversionoftheNSAAcan beusedinboysfromtheageof3yearstoobtaininformationonhowyoungDMDboys acquirenewabilitiesandhowthiscorrelateswiththeirpeers.Pubblicazioni consigliate
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