Data di Pubblicazione:
2016
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.
Tipologia CRIS:
14.a.1 Articolo su rivista
Keywords:
Medicine (all); Biochemistry, Genetics and Molecular Biology (all); Agricultural and Biological Sciences (all)
Elenco autori:
Mercuri, Eugenio; Coratti, Giorgia; Messina, Sonia; Ricotti, Valeria; Baranello, Giovanni; D'Amico, Adele; Pera, Maria Carmela; Albamonte, Emilio; Sivo, Serena; Mazzone, Elena Stacy; Arnoldi, Maria Teresa; Fanelli, Lavinia; De Sanctis, Roberto; Romeo, Domenico M.; Vita, Gian Luca; Battini, Roberta; Bertini, Enrico; Muntoni, Francesco; Pane, Marika
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