{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T08:10:16Z","timestamp":1741853416724,"version":"3.38.0"},"reference-count":24,"publisher":"Springer Science and Business Media LLC","issue":"2","license":[{"start":{"date-parts":[[2025,1,15]],"date-time":"2025-01-15T00:00:00Z","timestamp":1736899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2025,1,15]],"date-time":"2025-01-15T00:00:00Z","timestamp":1736899200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"name":"Biohaven Pharceuticals, Inc."}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Neurol Ther"],"published-print":{"date-parts":[[2025,4]]},"DOI":"10.1007\/s40120-024-00704-8","type":"journal-article","created":{"date-parts":[[2025,1,15]],"date-time":"2025-01-15T12:30:21Z","timestamp":1736944221000},"page":"547-563","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Content Validity of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia"],"prefix":"10.1007","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7389-3354","authenticated-orcid":false,"given":"Michele","family":"Potashman","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katja","family":"Rudell","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Naomi","family":"Suminski","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rinchen","family":"Doma","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maggie","family":"Heinrich","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Linda","family":"Abetz-Webb","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Melissa Wolfe","family":"Beiner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vlad","family":"Coric","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Liana S.","family":"Rosenthal","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sheng-Han","family":"Kuo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Theresa","family":"Zesiewicz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Terry D.","family":"Fife","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bart","family":"van de Warrenburg","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giovanni","family":"Ristori","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Matthis","family":"Synofzik","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sub","family":"Subramony","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Susan","family":"Perlman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeremy D.","family":"Schmahmann","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gil","family":"L\u2019Italien","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2025,1,15]]},"reference":[{"issue":"2","key":"704_CR1","doi-asserted-by":"publisher","first-page":"101","DOI":"10.1080\/14737175.2022.2029703","volume":"22","author":"SD Ghanekar","year":"2022","unstructured":"Ghanekar SD, Kuo SH, Staffetti JS, Zesiewicz TA. Current and emerging treatment modalities for spinocerebellar ataxias. Expert Rev Neurother. 2022;22(2):101\u201314.","journal-title":"Expert Rev Neurother"},{"issue":"2","key":"704_CR2","doi-asserted-by":"publisher","first-page":"533","DOI":"10.1007\/s00415-018-9076-4","volume":"266","author":"R Sullivan","year":"2019","unstructured":"Sullivan R, Yau WY, O\u2019Connor E, Houlden H. Spinocerebellar ataxia: an update. J Neurol. 2019;266(2):533\u201344.","journal-title":"J Neurol"},{"issue":"10","key":"704_CR3","doi-asserted-by":"publisher","first-page":"1833","DOI":"10.1002\/acn3.51875","volume":"10","author":"H Jacobi","year":"2023","unstructured":"Jacobi H, Schaprian T, Schmitz-H\u00fcbsch T, et al. Disease progression of spinocerebellar ataxia types 1, 2, 3 and 6 before and after ataxia onset. Ann Clin Transl Neurol. 2023;10(10):1833\u201343.","journal-title":"Ann Clin Transl Neurol"},{"key":"704_CR4","unstructured":"Perlman S. Hereditary ataxia overview. 1998. Seattle (WA): University of Washington, Seattle. https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK1138\/. Accessed May 16, 2024."},{"issue":"4","key":"704_CR5","doi-asserted-by":"publisher","first-page":"444","DOI":"10.1136\/jnnp-2020-325092","volume":"92","author":"R Sullivan","year":"2021","unstructured":"Sullivan R, Yau WY, Chelban V, et al. RFC1-related ataxia is a mimic of early multiple system atrophy. J Neurol Neurosurg Psychiatry. 2021;92(4):444\u20136.","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"10","key":"704_CR6","doi-asserted-by":"publisher","first-page":"4144","DOI":"10.1093\/brain\/awad157","volume":"146","author":"C Wilke","year":"2023","unstructured":"Wilke C, Pellerin D, Mengel D, et al. GAA-FGF14 ataxia (SCA27B): phenotypic profile, natural history progression and 4-aminopyridine treatment response. Brain. 2023;146(10):4144\u201357.","journal-title":"Brain"},{"issue":"8","key":"704_CR7","doi-asserted-by":"publisher","first-page":"1557","DOI":"10.1002\/mds.29559","volume":"38","author":"H Hengel","year":"2023","unstructured":"Hengel H, Pellerin D, Wilke C, et al. As frequent as polyglutamine spinocerebellar ataxias: SCA27B in a large German autosomal dominant ataxia cohort. Mov Disord. 2023;38(8):1557\u20138.","journal-title":"Mov Disord"},{"issue":"5","key":"704_CR8","doi-asserted-by":"publisher","first-page":"587","DOI":"10.1002\/mds.22740","volume":"25","author":"T Schmitz-H\u00fcbsch","year":"2010","unstructured":"Schmitz-H\u00fcbsch T, Coudert M, Giunti P, et al. Self-rated health status in spinocerebellar ataxia\u2013results from a European multicenter study. Mov Disord. 2010;25(5):587\u201395.","journal-title":"Mov Disord"},{"issue":"4","key":"704_CR9","doi-asserted-by":"publisher","first-page":"1466","DOI":"10.1007\/s12311-024-01657-2","volume":"23","author":"N Weber","year":"2024","unstructured":"Weber N, Buchholz M, R\u00e4dke A, et al. Factors influencing health-related quality of life of patients with spinocerebellar ataxia. Cerebellum. 2024;23(4):1466\u201377.","journal-title":"Cerebellum"},{"issue":"3","key":"704_CR10","doi-asserted-by":"publisher","first-page":"1020","DOI":"10.1007\/s12311-023-01597-3","volume":"23","author":"M Buchholz","year":"2023","unstructured":"Buchholz M, Weber N, R\u00e4dke A, et al. Health-related quality of life in patients with spinocerebellar ataxia: a validation study of the EQ-5D-3L. Cerebellum. 2023;23(3):1020\u201330.","journal-title":"Cerebellum"},{"issue":"4","key":"704_CR11","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1016\/S1474-4422(18)30042-5","volume":"17","author":"A Diallo","year":"2018","unstructured":"Diallo A, Jacobi H, Cook A, et al. Survival in patients with spinocerebellar ataxia types 1, 2, 3, and 6 (EUROSCA): a longitudinal cohort study. Lancet Neurol. 2018;17(4):327\u201334.","journal-title":"Lancet Neurol"},{"issue":"2","key":"704_CR12","doi-asserted-by":"publisher","first-page":"202","DOI":"10.1002\/acn3.156","volume":"2","author":"M-L Monin","year":"2015","unstructured":"Monin M-L, Tezenas du Montcel S, Marelli C, et al. Survival and severity in dominant cerebellar ataxias. Ann Clin Transl Neurol. 2015;2(2):202\u20137.","journal-title":"Ann Clin Transl Neurol"},{"issue":"11","key":"704_CR13","doi-asserted-by":"publisher","first-page":"1101","DOI":"10.1016\/S1474-4422(15)00202-1","volume":"14","author":"H Jacobi","year":"2015","unstructured":"Jacobi H, du Montcel ST, Bauer P, et al. Long-term disease progression in spinocerebellar ataxia types 1, 2, 3, and 6: a longitudinal cohort study. Lancet Neurol. 2015;14(11):1101\u20138.","journal-title":"Lancet Neurol"},{"issue":"6","key":"704_CR14","doi-asserted-by":"publisher","first-page":"1257","DOI":"10.1007\/s12311-022-01494-1","volume":"22","author":"MH Potashman","year":"2023","unstructured":"Potashman MH, Mize ML, Beiner MW, Pierce S, Coric V, Schmahmann JD. Ataxia rating scales reflect patient experience: an examination of the relationship between clinician assessments of cerebellar ataxia and patient-reported outcomes. Cerebellum. 2023;22(6):1257\u201373.","journal-title":"Cerebellum"},{"key":"704_CR15","doi-asserted-by":"publisher","first-page":"902","DOI":"10.3389\/fneur.2020.00902","volume":"11","author":"CC Lin","year":"2020","unstructured":"Lin CC, Ashizawa T, Kuo SH. Collaborative efforts for spinocerebellar ataxia research in the United States: CRC-SCA and READISCA. Front Neurol. 2020;11:902.","journal-title":"Front Neurol"},{"issue":"2","key":"704_CR16","doi-asserted-by":"publisher","first-page":"137","DOI":"10.1590\/0004-282x-anp-2020-0580","volume":"80","author":"GCD Cruz","year":"2022","unstructured":"Cruz GCD, Zonta MB, Munhoz RP, et al. Functionality and disease severity in spinocerebellar ataxias. Arq Neuropsiquiatr. 2022;80(2):137\u201344.","journal-title":"Arq Neuropsiquiatr"},{"issue":"11","key":"704_CR17","doi-asserted-by":"publisher","first-page":"1711","DOI":"10.1212\/01.wnl.0000218155.46739.90","volume":"66","author":"DR Lynch","year":"2006","unstructured":"Lynch DR, Farmer JM, Tsou AY, et al. Measuring Friedreich ataxia: complementary features of examination and performance measures. Neurology. 2006;66(11):1711\u20136.","journal-title":"Neurology"},{"issue":"4","key":"704_CR18","doi-asserted-by":"publisher","first-page":"426","DOI":"10.1002\/mds.22912","volume":"25","author":"LS Friedman","year":"2010","unstructured":"Friedman LS, Farmer JM, Perlman S, et al. Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design. Mov Disord. 2010;25(4):426\u201332.","journal-title":"Mov Disord"},{"issue":"3","key":"704_CR19","doi-asserted-by":"publisher","first-page":"924","DOI":"10.1007\/s12311-023-01547-z","volume":"23","author":"T Klockgether","year":"2024","unstructured":"Klockgether T, Synofzik M, Alhusaini S, et al. Consensus recommendations for clinical outcome assessments and registry development in ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance. Cerebellum. 2024;23(3):924\u201330.","journal-title":"Cerebellum"},{"issue":"6","key":"704_CR20","doi-asserted-by":"publisher","first-page":"965","DOI":"10.1002\/mds.29788","volume":"39","author":"A Trasch\u00fctz","year":"2024","unstructured":"Trasch\u00fctz A, Fleszar Z, Hengel H, et al. FARS-ADL across ataxias: construct validity, sensitivity to change, and minimal important change. Mov Disord. 2024;39(6):965\u201374.","journal-title":"Mov Disord"},{"issue":"5","key":"704_CR21","doi-asserted-by":"publisher","first-page":"2012","DOI":"10.1007\/s12311-024-01700-2","volume":"23","author":"M Potashman","year":"2024","unstructured":"Potashman M, Rudell K, Pavisic I, et al. Content validity of the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) instrument in spinocerebellar ataxia. Cerebellum. 2024;23(5):2012\u201327.","journal-title":"Cerebellum"},{"issue":"10","key":"704_CR22","doi-asserted-by":"publisher","first-page":"2367","DOI":"10.1002\/mds.28670","volume":"36","author":"JD Schmahmann","year":"2021","unstructured":"Schmahmann JD, Pierce S, MacMore J, L\u2019Italien GJ. Development and validation of a patient-reported outcome measure of ataxia. Mov Disord. 2021;36(10):2367\u201377.","journal-title":"Mov Disord"},{"issue":"8","key":"704_CR23","doi-asserted-by":"publisher","first-page":"967","DOI":"10.1016\/j.jval.2011.06.014","volume":"14","author":"DL Patrick","year":"2011","unstructured":"Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity\u2013establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 1\u2013eliciting concepts for a new PRO instrument. Value Health. 2011;14(8):967\u201377.","journal-title":"Value Health"},{"key":"704_CR24","volume-title":"Discovery of grounded theory: strategies for qualitative research","author":"B Glaser","year":"1999","unstructured":"Glaser B, Strauss A. Discovery of grounded theory: strategies for qualitative research. 1st ed. New York: Routledge; 1999.","edition":"1"}],"container-title":["Neurology and Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-024-00704-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40120-024-00704-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-024-00704-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T07:30:48Z","timestamp":1741851048000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40120-024-00704-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1,15]]},"references-count":24,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,4]]}},"alternative-id":["704"],"URL":"https:\/\/doi.org\/10.1007\/s40120-024-00704-8","relation":{},"ISSN":["2193-8253","2193-6536"],"issn-type":[{"type":"print","value":"2193-8253"},{"type":"electronic","value":"2193-6536"}],"subject":[],"published":{"date-parts":[[2025,1,15]]},"assertion":[{"value":"13 November 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"18 December 2024","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"15 January 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Michele Potashman, Melissa Wolfe Beiner, Vlad Coric, and Gil L\u2019Italien are employed by, and own shares in, Biohaven Pharmaceuticals, Inc. Katja Rudell, Naomi Suminski, Rinchen Doma, and Maggie Heinrich are employees of Parexel and were commissioned by Biohaven Pharmaceuticals, Inc. to conduct the study. Sheng-Han Kuo, Theresa Zesiwicz, Bart van de Warrenburg, Liana S. Rosenthal, Terry D. Fife, Giovanni Ristori, Matthis Synofzik, and Susan Perlman received consultancy fees from Biohaven Pharmaceuticals, Inc. for participating in the interviews. Linda Abetz-Webb received consultancy fees from Parexel for this study. Sheng-Han Kuo has received consultancy fees from Biohaven Pharmaceuticals, Inc., Praxis Precision Medicines, and Sage Therapeutics. Liana S. Rosenthal receives salary support for her role as the site Principal Investigator for research studies with Biohaven Pharmaceuticals, Inc., Pfizer, and EIP Pharma, and for serving on the Clinical Events Committee for a research study with functional neuromodulation. She also serves on the steering committees for the Parkinson Study Group\u2019s research study with both UCB Pharmaceuticals and Bial Pharmaceuticals. Dr. Rosenthal also served on an advisory board for Reata Pharmaceuticals, Biogen Pharmaceuticals, Biohaven Pharmaceuticals, Inc., and as a one-time consultant for UCB Pharmaceuticals. Theresa Zesiwicz has received personal compensation for serving on the advisory boards of Boston Scientific, Reata Pharmaceuticals, and Steminent Biotherapeutics; and received personal compensation as senior editor for Neurodegenerative Disease Management and as a consultant for Steminent Biotherapeutics. Royalties: royalty payments as co-inventor of varenicline for treating imbalance and nonataxic imbalance. Grants: research grant support as Principal Investigator for studies from AbbVie, Biogen Pharmaceuticals, Biohaven Pharmaceuticals, Inc., Boston Scientific, Bukwang Pharmaceuticals, Cala Health, Cavion, Friedreich\u2019s Ataxia Research Alliance; Houston Methodist Research Institute, National Institutes of Health, Retrotope, and Takeda Development Center Americas. Bart van de Warrenburg has served on advisory boards and\/or as consultant for Servier, VICO Therapeutics, Biohaven Pharmaceuticals, Inc., and uniQure. Royalties: BSL\u2014Springer Nature. Grants: Radboud University Medical Center, ZonMw, Gossweiler Foundation, Hersenstichting, NWO, and Christina Foundation. Jeremy D. Schmahmann has served on the Editorial Board for The Cerebellum since 1999. Consultancy: Biohaven Pharmaceuticals, Inc. Site Principal Investigator: Biohaven Pharmaceuticals, Inc. clinical trials in ataxia and multiple system atrophy. Research support, commercial entities: Biohaven Pharmaceuticals, Inc. support of clinical trials. Research support, academic entities: National Ataxia Foundation. Research support, foundations, and societies: National Ataxia Foundation, 2019, Principal Investigator license fee payments. Technology or inventions: Brief Ataxia Rating Scale (BARS) and Brief Ataxia Rating Scale revised (BARS2), copyright held by The General Hospital Corporation; Cerebellar Cognitive Affective\/Schmahmann syndrome Scale, copyright held by The General Hospital Corporation; Patient-Reported Outcome Measure of Ataxia, copyright held by The General Hospital Corporation; and Cerebellar Neuropsychiatric Rating Scale, copyright held by The General Hospital Corporation. Matthis Synofzik has received consultancy honoraria from Ionis, UCB Pharmaceuticals, Prevail, Orphazyme, Biogen Pharmaceuticals, Servier, Reata Pharmaceuticals, Gen-Orph, AviadoBio, Zevra, Lilly, and Solaxa, all unrelated to the present manuscript.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"The study (BHV-4157-SCA-VAL) was approved by a centralized independent institutional review board (IRB; Salus Institutional Review Board, Austin, TX, USA) on 15 July 2022. IRB approval was not required for HCP interviews conducted in the United States or Europe. HCPs received consultancy fees for participating in the interviews. All eligible individuals with SCA and the HCPs provided informed consent to participate in the interviews and could withdraw at any time. The study was conducted in accordance with the 1964 Declaration of Helsinki.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}