Journal article
Goal-Directed Rehabilitation Versus Standard Care for Individuals with Hereditary Cerebellar Ataxia: A Multicenter, Single-Blind, Randomized Controlled Superiority Trial
SC Milne, M Roberts, S Williams, J Chua, AC Grootendorst, G Agostinelli, AC Grobler, HL Ross, A Robinson, K Grove, G Modderman, A Price, M Thomson, L Massey, C Liang, KR Kumar, K Dalziel, J Burns, CM Sue, PN Pathirana Show all
Annals of Neurology | WILEY | Published : 2025
DOI: 10.1002/ana.27130
Abstract
Objective: 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-week goal-directed rehabilitation program compared with 30 weeks of standard care on function, ataxia, health-related quality of life, and balance in individuals with an HCA. Methods: Individuals with an autosomal dominant or recessive ataxia (aged ≥15 years) were enrolled at 5 sites in Australia. Participants were randomized (1:1) to receive rehabilitation (6 weeks of outpatient physiotherapy followed by a 24-week home exercise program) (n = 39) or continued their usual activity ..
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Funding Acknowledgements
This work was supported by an Australian Government Medical Research Future Fund (MRFF) Lifting Clinical Trials and Registries Capacity Program Grant [APP1152226] and a Rebecca L Cooper grant [PG2018135] and a MRFF Career Development Fellowship [APP1143098; L.A.C.]. The authors thank all the participants who gave their time for this study and thank the Cerebellar Ataxia Australia Association and the Friedreich Ataxia Network for their assistance in recruitment and the MJD Foundation for their support.