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dc.contributor.authorMercuri, Eugenio
dc.contributor.authorFinkel, Richard
dc.contributor.authorMontes, Jacqueline
dc.contributor.authorMazzone, Elena S
dc.contributor.authorSormani, Maria Pia
dc.contributor.authorMain, Marion
dc.contributor.authorRamsey, Danielle
dc.contributor.authorMayhew, Anna
dc.contributor.authorGlanzman, Allan M
dc.contributor.authorDunaway, Sally
dc.contributor.authorSalazar, Rachel
dc.contributor.authorPasternak, Amy
dc.contributor.authorQuigley, Janet
dc.contributor.authorPane, Marika
dc.contributor.authorPera, Maria Carmela
dc.contributor.authorScoto, Mariacristina
dc.contributor.authorMessina, Sonia
dc.contributor.authorSframeli, Maria
dc.contributor.authorVita, Gian Luca
dc.contributor.authorD'Amico, Adele
dc.contributor.authorvan den Hauwe, Marleen
dc.contributor.authorSivo, Serena
dc.contributor.authorGoemans, Nathalie
dc.contributor.authorKaufmann, Petra
dc.contributor.authorDarras, Basil T
dc.contributor.authorBertini, Enrico
dc.contributor.authorMuntoni, Francesco
dc.contributor.authorDe Vivo, Darryl C
dc.date.accessioned2018-02-06T17:28:31Z
dc.date.available2018-02-06T17:28:31Z
dc.date.issued2016-02
dc.identifier.citationMercuri , E , Finkel , R , Montes , J , Mazzone , E S , Sormani , M P , Main , M , Ramsey , D , Mayhew , A , Glanzman , A M , Dunaway , S , Salazar , R , Pasternak , A , Quigley , J , Pane , M , Pera , M C , Scoto , M , Messina , S , Sframeli , M , Vita , G L , D'Amico , A , van den Hauwe , M , Sivo , S , Goemans , N , Kaufmann , P , Darras , B T , Bertini , E , Muntoni , F & De Vivo , D C 2016 , ' Patterns of disease progression in type 2 and 3 SMA : Implications for clinical trials ' , Neuromuscular disorders : NMD , vol. 26 , no. 2 , pp. 126-31 . https://doi.org/10.1016/j.nmd.2015.10.006
dc.identifier.issn0960-8966
dc.identifier.otherPURE: 11163480
dc.identifier.otherPURE UUID: def639e9-1a0b-40d2-9d9c-4bbb69b0af83
dc.identifier.otherPubMed: 26776503
dc.identifier.otherPubMedCentral: PMC4762230
dc.identifier.otherScopus: 84956949641
dc.identifier.urihttp://hdl.handle.net/2299/19729
dc.descriptionThis is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License CC BY NC-ND 4.0 ( http://creativecommons.org/licenses/by-nc-nd/4.0/ ), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited, and is not altered, transformed, or built upon in any way.
dc.description.abstractThe aim of the study was to establish 12-month changes in the Hammersmith Functional motor scale in a large cohort of SMA patients, to identify patterns of disease progression and the effect of different variables. 268 patients were included in this multicentric study. Their age ranged between 2.5 and 55.5 years at baseline, 68 were ambulant and 200 non-ambulant. The baseline scores ranged between 0 and 66 (mean 23.91, SD 20.09). The 12-month change was between -14 and +9 (mean -0.56, SD 2.72). Of the 268 patients, 206 (76.86%) had changes between -2 and +2 points. Ambulant and non-ambulant subjects had a different relationship between baseline values and age (p for age X ambulation interaction = 0.007). There was no association with age in ambulant subjects, while there was a significant heterogeneity at different age for non-ambulant patients (p < 0.001). The 12-month change (adjusted for baseline) was not associated with age in ambulant patients (p = 0.34), but it was significantly different among various age groups in non-ambulant patients. Our results suggest that there are different profiles of progression in ambulant and non-ambulant patients, and that age may play an important role in the progression of non-ambulant patients.en
dc.format.extent6
dc.language.isoeng
dc.relation.ispartofNeuromuscular disorders : NMD
dc.rightsOpen
dc.subjectAdolescent
dc.subjectAdult
dc.subjectChild
dc.subjectChild, Preschool
dc.subjectClinical Trials as Topic
dc.subjectDisease Progression
dc.subjectFemale
dc.subjectHumans
dc.subjectMale
dc.subjectMiddle Aged
dc.subjectMobility Limitation
dc.subjectRetrospective Studies
dc.subjectSpinal Muscular Atrophies of Childhood
dc.subjectYoung Adult
dc.subjectJournal Article
dc.subjectMulticenter Study
dc.subjectResearch Support, Non-U.S. Gov't
dc.titlePatterns of disease progression in type 2 and 3 SMA : Implications for clinical trialsen
dc.contributor.institutionSchool of Health and Social Work
dc.description.statusPeer reviewed
dc.relation.schoolSchool of Health and Social Work
dc.description.versiontypeFinal Published version
dcterms.dateAccepted2016-02
rioxxterms.versionVoR
rioxxterms.versionofrecordhttps://doi.org/10.1016/j.nmd.2015.10.006
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
rioxxterms.typeJournal Article/Review
herts.rights.accesstypeOpen


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