{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T00:20:03Z","timestamp":1793146803459,"version":"4.5.0"},"reference-count":31,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2023,8,28]],"date-time":"2023-08-28T00:00:00Z","timestamp":1693180800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003196","name":"Ministero della Salute","doi-asserted-by":"publisher","award":["GrantRF\u20102019\u201012368918(toCM)"],"award-info":[{"award-number":["GrantRF\u20102019\u201012368918(toCM)"]}],"id":[{"id":"10.13039\/501100003196","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000780","name":"European Commission","doi-asserted-by":"publisher","award":["HEALTH\u2010F2\u20102010\u2010242193"],"award-info":[{"award-number":["HEALTH\u2010F2\u20102010\u2010242193"]}],"id":[{"id":"10.13039\/501100000780","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100018556","name":"Voyager Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018556","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The Scale for Assessment and Rating of Ataxia (SARA) is widely used in different types of ataxias and has been chosen as the primary outcome measure in the European natural history study for Friedreich ataxia (FA).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>To assess distribution and longitudinal changes of SARA scores and its single items, we analyzed SARA scores of 502 patients with typical\u2010onset FA (&lt;25\u2009years) participating in the 4\u2010year prospective European FA Consortium for Translational Studies (EFACTS). Pattern of disease progression was determined using linear mixed\u2010effects regression models. The chosen statistical model was re\u2010fitted in order to estimate parameters and predict disease progression. Median time\u2010to\u2010change and rate of score progression were estimated using the Kaplan\u2013Meier method and weighted linear regression models, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>SARA score at study enrollment and age at onset were the major predictive factors of total score progression during the 4\u2010year follow\u2010up. To a less extent, age at evaluation also influenced the speed of SARA progression, while disease duration did not improve the prediction of the statistical model. Temporal dynamics of total SARA and items showed a great variability in the speed of score increase during disease progression. Gait item had the highest annual progression rate, with median time for one\u2010point score increase of 1 to 2\u2009years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Analyses of statistical properties of SARA suggest a variable sensitivity of the scale at different disease stages, and provide important information for population selection and result interpretation in future clinical trials.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.51886","type":"journal-article","created":{"date-parts":[[2023,8,29]],"date-time":"2023-08-29T05:29:10Z","timestamp":1693286950000},"page":"2000-2012","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Longitudinal changes of <scp>SARA<\/scp> scale in Friedreich ataxia: Strong influence of baseline score and age at onset"],"prefix":"10.1002","volume":"10","author":[{"given":"Luca","family":"Porcu","sequence":"first","affiliation":[{"name":"Cancer Research UK Cambridge Institute University of Cambridge  Cambridge UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mario","family":"Fichera","sequence":"additional","affiliation":[{"name":"Unit of Medical Genetics and Neurogenetics Fondazione IRCCS Istituto Neurologico Carlo Besta  Milan 20133 Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorenzo","family":"Nanetti","sequence":"additional","affiliation":[{"name":"Unit of Medical Genetics and Neurogenetics Fondazione IRCCS Istituto Neurologico Carlo Besta  Milan 20133 Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eliana","family":"Rulli","sequence":"additional","affiliation":[{"name":"Laboratory of Methodology for Clinical Research, Oncology Department Istituto di Ricerche Farmacologiche Mario Negri IRCCS  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paola","family":"Giunti","sequence":"additional","affiliation":[{"name":"Department of Clinical and Movement Neurosciences Ataxia Centre, UCL\u2010Queen Square Institute of Neurology  London WC1N 3BG UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael H.","family":"Parkinson","sequence":"additional","affiliation":[{"name":"Department of Clinical and Movement Neurosciences Ataxia Centre, UCL\u2010Queen Square Institute of Neurology  London WC1N 3BG UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"Durr","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, Paris Brain Institute (ICM Institut du Cerveau), AP\u2010HP, INSERM, CNRS University Hospital Piti\u00e9\u2010Salp\u00eatri\u00e8re  Paris 75646 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire","family":"Ewenczyk","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9, Paris Brain Institute (ICM Institut du Cerveau), AP\u2010HP, INSERM, CNRS University Hospital Piti\u00e9\u2010Salp\u00eatri\u00e8re  Paris 75646 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"Boesch","sequence":"additional","affiliation":[{"name":"Department of Neurology Medical University Innsbruck  Innsbruck 6020 Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang","family":"Nachbauer","sequence":"additional","affiliation":[{"name":"Department of Neurology Medical University Innsbruck  Innsbruck 6020 Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0217-8630","authenticated-orcid":false,"given":"Elisabetta","family":"Indelicato","sequence":"additional","affiliation":[{"name":"Department of Neurology Medical University Innsbruck  Innsbruck 6020 Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Klopstock","sequence":"additional","affiliation":[{"name":"Department of Neurology Friedrich Baur Institute, University Hospital, LMU  Munich 80336 Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE)  Munich 81377 Germany"},{"name":"Munich Cluster for Systems Neurology (SyNergy)  Munich 81377 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4234-8393","authenticated-orcid":false,"given":"Claudia","family":"Stendel","sequence":"additional","affiliation":[{"name":"Department of Neurology Friedrich Baur Institute, University Hospital, LMU  Munich 80336 Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE)  Munich 81377 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francisco Javier","family":"Rodr\u00edguez de Rivera","sequence":"additional","affiliation":[{"name":"Reference Unit of Hereditary Ataxias and Paraplegias, Department of Neurology IdiPAZ, Hospital Universitario La Paz  Madrid 28046 Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ludger","family":"Sch\u00f6ls","sequence":"additional","affiliation":[{"name":"Department of Neurology and Hertie\u2010Institute for Clinical Brain Research University of T\u00fcbingen  T\u00fcbingen 72076 Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE)  T\u00fcbingen 72076 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zofia","family":"Fleszar","sequence":"additional","affiliation":[{"name":"Department of Neurology and Hertie\u2010Institute for Clinical Brain Research University of T\u00fcbingen  T\u00fcbingen 72076 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Giordano","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospital of Bonn  Bonn 53127 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire","family":"Didszun","sequence":"additional","affiliation":[{"name":"Department of Neurology RWTH Aachen University  Aachen 52074 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Castaldo","sequence":"additional","affiliation":[{"name":"Unit of Medical Genetics and Neurogenetics Fondazione IRCCS Istituto Neurologico Carlo Besta  Milan 20133 Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Myriam","family":"Rai","sequence":"additional","affiliation":[{"name":"Laboratory of Experimental Neurology Universit\u00e9 Libre de Bruxelles  Brussels 1070 Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Klockgether","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospital of Bonn  Bonn 53127 Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE)  Bonn 53127 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimo","family":"Pandolfo","sequence":"additional","affiliation":[{"name":"Laboratory of Experimental Neurology Universit\u00e9 Libre de Bruxelles  Brussels 1070 Belgium"},{"name":"Department of Neurology and Neurosurgery McGill University  Montreal QC H3A 0G4 Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8903-0593","authenticated-orcid":false,"given":"J\u00f6rg B.","family":"Schulz","sequence":"additional","affiliation":[{"name":"Department of Neurology RWTH Aachen University  Aachen 52074 Germany"},{"name":"JARA Brain Institute Molecular Neuroscience and Neuroimaging, Research Centre J\u00fclich and RWTH Aachen University  Aachen 52056 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9730-9228","authenticated-orcid":false,"given":"Kathrin","family":"Reetz","sequence":"additional","affiliation":[{"name":"Department of Neurology RWTH Aachen University  Aachen 52074 Germany"},{"name":"JARA Brain Institute Molecular Neuroscience and Neuroimaging, Research Centre J\u00fclich and RWTH Aachen University  Aachen 52056 Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2405-3564","authenticated-orcid":false,"given":"Caterina","family":"Mariotti","sequence":"additional","affiliation":[{"name":"Unit of Medical Genetics and Neurogenetics Fondazione IRCCS Istituto Neurologico Carlo Besta  Milan 20133 Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"for the EFACTS Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,8,28]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000219042.60538.92"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(15)00202-1"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2019-322168"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1212\/NXG.0000000000200034"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199610173351601"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006121"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/6.11.1771"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/104.3.589"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.26382"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.22912"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(14)70321-7"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000156802.15466.79"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.332"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000200913"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51118"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1212\/NXG.0000000000000371"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2019.104606"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25934"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29286"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181d1a6c9"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2017-316964"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29255"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29135"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.23879"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(16)30287-3"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00027-2"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1212\/NXG.0000000000000415"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-022-23666-z"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-4614-7138-7"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-3-319-24277-4"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29370"}],"container-title":["Annals of Clinical and Translational Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/acn3.51886","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,15]],"date-time":"2023-11-15T21:22:40Z","timestamp":1700083360000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/acn3.51886"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,8,28]]},"references-count":31,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2023,11]]}},"alternative-id":["10.1002\/acn3.51886"],"URL":"https:\/\/doi.org\/10.1002\/acn3.51886","archive":["Portico"],"relation":{},"ISSN":["2328-9503","2328-9503"],"issn-type":[{"value":"2328-9503","type":"print"},{"value":"2328-9503","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,8,28]]},"assertion":[{"value":"2023-05-23","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-08-10","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-08-28","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}