{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,21]],"date-time":"2026-04-21T04:30:30Z","timestamp":1776745830232,"version":"3.51.2"},"reference-count":41,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,11,9]],"date-time":"2024-11-09T00:00:00Z","timestamp":1731110400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100003921","name":"Department of Health and Aged Care, Australian Government","doi-asserted-by":"publisher","award":["APP1143098"],"award-info":[{"award-number":["APP1143098"]}],"id":[{"id":"10.13039\/501100003921","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003921","name":"Department of Health and Aged Care, Australian Government","doi-asserted-by":"publisher","award":["APP1152226"],"award-info":[{"award-number":["APP1152226"]}],"id":[{"id":"10.13039\/501100003921","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Annals of Neurology"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:sec><jats:title>Objective<\/jats:title><jats:p>Rehabilitation is thought to reduce ataxia severity in individuals with hereditary cerebellar ataxia (HCA). This multicenter, randomized controlled superiority trial aimed to examine the efficacy of a 30\u2010week goal\u2010directed rehabilitation program compared with 30\u2009weeks of standard care on function, ataxia, health\u2010related quality of life, and balance in individuals with an HCA.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Individuals with an autosomal dominant or recessive ataxia (aged \u226515\u2009years) were enrolled at 5 sites in Australia. Participants were randomized (1:1) to receive rehabilitation (6\u2009weeks of outpatient physiotherapy followed by a 24\u2010week home exercise program) (n\u2009=\u200939) or continued their usual activity (n\u2009=\u200937). The primary outcome measure was the motor domain of the Functional Independence Measure (mFIM) at 7\u2009weeks. Secondary outcomes included the Scale for the Assessment and Rating of Ataxia (SARA) and the SF\u201036v2, assessed at 7, 18, and 30\u2009weeks. Outcome assessors were blinded to treatment allocation.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Seventy\u2010one participants (rehabilitation, 37; standard\u2010care, 34) were included in the intention\u2010to\u2010treat analysis. At 7\u2009weeks, mFIM (mean difference 2.26, 95% confidence interval [CI]: 0.26 to 4.26, <jats:italic>p<\/jats:italic>\u2009=\u20090.028) and SARA (\u22121.21, 95% CI: \u22122.32 to \u22120.11, <jats:italic>p<\/jats:italic>\u2009=\u20090.032) scores improved after rehabilitation compared with standard care. Compared with standard care, rehabilitation improved SARA scores at 30\u2009weeks (mean difference \u22121.51, 95% CI: \u22122.76 to \u22120.27, <jats:italic>p<\/jats:italic>\u2009=\u20090.017), but not mFIM scores (1.74, 95% CI: \u22120.32 to 3.81, <jats:italic>p<\/jats:italic>\u2009=\u20090.098). Frequent adverse events in both groups were fatigue, pain, and falls.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Goal\u2010directed rehabilitation improved function at 7\u2009weeks, with improvement in ataxia and health\u2010related quality of life maintained at 30\u2009weeks in individuals with HCA, beyond that of standard care. ANN NEUROL 2025;97:409\u2013424<\/jats:p><\/jats:sec>","DOI":"10.1002\/ana.27130","type":"journal-article","created":{"date-parts":[[2024,11,9]],"date-time":"2024-11-09T11:14:15Z","timestamp":1731150855000},"page":"409-424","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Goal\u2010Directed Rehabilitation Versus Standard Care for Individuals with Hereditary Cerebellar Ataxia: A Multicenter, Single\u2010Blind, Randomized Controlled Superiority Trial"],"prefix":"10.1002","volume":"97","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9406-8609","authenticated-orcid":false,"given":"Sarah C","family":"Milne","sequence":"first","affiliation":[{"name":"Bruce Lefroy Centre for Genetic Health Research Murdoch Children's Research Institute  Parkville Australia"},{"name":"Physiotherapy Department Monash Health  Cheltenham Australia"},{"name":"School of Primary and Allied Health Care Monash University  Frankston Australia"},{"name":"Department of Pediatrics The University of Melbourne  Parkville Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Melissa","family":"Roberts","sequence":"additional","affiliation":[{"name":"Bruce Lefroy Centre for Genetic Health Research Murdoch Children's Research Institute  Parkville Australia"},{"name":"Physiotherapy Department Monash Health  Cheltenham Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shannon","family":"Williams","sequence":"additional","affiliation":[{"name":"Physiotherapy Department Sir Charles Gairdner Hospital  Nedlands Australia"},{"name":"Physiotherapy Department Royal Perth Hospital  Perth Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jillian","family":"Chua","sequence":"additional","affiliation":[{"name":"Ryde Hospital  Eastwood Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alison C","family":"Grootendorst","sequence":"additional","affiliation":[{"name":"MJD Foundation  Darwin Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Genevieve","family":"Agostinelli","sequence":"additional","affiliation":[{"name":"MJD Foundation  Darwin Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anneke C","family":"Grobler","sequence":"additional","affiliation":[{"name":"Department of Pediatrics The University of Melbourne  Parkville Australia"},{"name":"Clinical Epidemiology and Biostatistics Unit Murdoch Children's Research Institute  Parkville Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hannah L","family":"Ross","sequence":"additional","affiliation":[{"name":"Physiotherapy Department Monash Health  Cheltenham Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amy","family":"Robinson","sequence":"additional","affiliation":[{"name":"Physiotherapy Department Royal North Shore Hospital  St Leonards Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristen","family":"Grove","sequence":"additional","affiliation":[{"name":"Physiotherapy Department Sir Charles Gairdner Hospital  Nedlands Australia"},{"name":"Physiotherapy Department Royal Perth Hospital  Perth Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gabrielle","family":"Modderman","sequence":"additional","affiliation":[{"name":"Rehabilitation Services Royal Darwin and Palmerston Regional Hospital  Darwin Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Annabel","family":"Price","sequence":"additional","affiliation":[{"name":"Physiotherapy Department Royal North Shore Hospital  St Leonards Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Megan","family":"Thomson","sequence":"additional","affiliation":[{"name":"Physiotherapy Department Monash Health  Cheltenham Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Libby","family":"Massey","sequence":"additional","affiliation":[{"name":"MJD Foundation  Darwin Australia"},{"name":"College of Public Health Medical and Veterinary Sciences James Cook University  Townsville Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christina","family":"Liang","sequence":"additional","affiliation":[{"name":"Kolling Institute of Medical Research University of Sydney  St Leonards Australia"},{"name":"Department of Neurology Royal North Shore Hospital  St Leonards Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3482-6962","authenticated-orcid":false,"given":"Kishore R","family":"Kumar","sequence":"additional","affiliation":[{"name":"Molecular Medicine Laboratory and Department of Neurology, Concord Repatriation General Hospital, Concord Clinical School The University of Syndey  Concord Australia"},{"name":"Translational Neurogenomics Group, Genomic and Inherited Disease Program Garvan Institute of Medical Research  Sydney Australia"},{"name":"St Vincent's Healthcare Clinical Campus UNSW Medicine &amp; Health, UNSW Sydney  Kensington Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kim","family":"Dalziel","sequence":"additional","affiliation":[{"name":"Melbourne School of Population and Global Health University of Melbourne  Parkville Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joshua","family":"Burns","sequence":"additional","affiliation":[{"name":"Disability Prevention Program St. Jude Children's Research Hospital  Memphis TN USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carolyn M","family":"Sue","sequence":"additional","affiliation":[{"name":"Centre for Neurodegeneration Neuroscience Research Australia  Sydney Australia"},{"name":"Faculty of Medicine and Health University of New South Wales  Sydney Australia"},{"name":"Department of Neurology Prince of Wales Hospital  Sydney Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pubudu N","family":"Pathirana","sequence":"additional","affiliation":[{"name":"School of Engineering Deakin University  Geelong Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Malcolm","family":"Horne","sequence":"additional","affiliation":[{"name":"Bionics Institute  East Melbourne Australia"},{"name":"Department of Medicine St Vincent's Hospital, The University of Melbourne  Parkville Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nikki","family":"Gelfand","sequence":"additional","affiliation":[{"name":"Monash Genetics, Monash Health  Clayton Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Helen","family":"Curd","sequence":"additional","affiliation":[{"name":"Monash Genetics, Monash Health  Clayton Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David","family":"Szmulewicz","sequence":"additional","affiliation":[{"name":"Bionics Institute  East Melbourne Australia"},{"name":"Balance Disorders and Ataxia Service Royal Victorian Eye and Ear Hospital  East Melbourne Australia"},{"name":"Monash Medical Centre Monash Health  Clayton Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Louise A","family":"Corben","sequence":"additional","affiliation":[{"name":"Bruce Lefroy Centre for Genetic Health Research Murdoch Children's Research Institute  Parkville Australia"},{"name":"Department of Pediatrics The University of Melbourne  Parkville Australia"},{"name":"Turner Institute for Brain and Mental Health Monash University  Clayton Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martin B","family":"Delatycki","sequence":"additional","affiliation":[{"name":"Bruce Lefroy Centre for Genetic Health Research Murdoch Children's Research Institute  Parkville Australia"},{"name":"Department of Pediatrics The University of Melbourne  Parkville Australia"},{"name":"Victorian Clinical Genetics Services  Melbourne Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,11,9]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1159\/000358801"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12311-022-01505-1"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12311-022-01494-1"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-02091-x"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nrl.2015.09.002"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25934"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-022-02568-3"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-019-1013-9"},{"key":"e_1_2_9_10_1","unstructured":"World Health Organization.Rehabilitation: World Health Organization 2023Available from.https:\/\/www.who.int\/news-room\/fact-sheets\/detail\/rehabilitation."},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-022-11383-6"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1177\/0269215517736903"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1177\/0269215520905073"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1177\/1545968311425918"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.27437"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.27676"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12311-019-1006-1"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.apmr.2015.01.021"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1089\/jicm.2021.0222"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1177\/0269215514539336"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181c33adf"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1155\/2014\/583507"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2020-040230"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2011.2504"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.apmr.2023.05.003"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.3390\/brainsci12080982"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.23222"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1177\/0269215509343846"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.1097\/00001199-199308020-00008"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0b013e3181a9f1d6"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181d1a6c9"},{"key":"e_1_2_9_32_1","unstructured":"Australian Institute of Health and Welfare.Rural and remote health: AIHW Australian Government 2024."},{"key":"e_1_2_9_33_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12311-022-01394-4"},{"key":"e_1_2_9_34_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(15)00201-X"},{"key":"e_1_2_9_35_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.apmr.2016.09.128"},{"key":"e_1_2_9_36_1","unstructured":"MaruishME.User's manual for the SF\u201036v2 health survey: quality metric incorporated 2011."},{"key":"e_1_2_9_37_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29788"},{"key":"e_1_2_9_38_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.humov.2017.01.010"},{"key":"e_1_2_9_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.socscimed.2019.06.004"},{"key":"e_1_2_9_40_1","doi-asserted-by":"publisher","DOI":"10.1093\/aje\/kwi054"},{"key":"e_1_2_9_41_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.26712"},{"key":"e_1_2_9_42_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(15)00202-1"}],"container-title":["Annals of Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ana.27130","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,17]],"date-time":"2025-02-17T12:32:38Z","timestamp":1739795558000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ana.27130"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,11,9]]},"references-count":41,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,3]]}},"alternative-id":["10.1002\/ana.27130"],"URL":"https:\/\/doi.org\/10.1002\/ana.27130","archive":["Portico"],"relation":{},"ISSN":["0364-5134","1531-8249"],"issn-type":[{"value":"0364-5134","type":"print"},{"value":"1531-8249","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,11,9]]},"assertion":[{"value":"2024-07-22","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-10-20","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-11-09","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}