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Article History

Received: 13 November 2024

Accepted: 18 December 2024

First Online: 15 January 2025

Declarations

:

: Michele Potashman, Melissa Wolfe Beiner, Vlad Coric, and Gil L’Italien 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’s 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’s 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—Springer 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.

: 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.