Longitudinal progression, metrics, age-dependence, and modifiers of ataxia severity in SCA27B: a multicentre study of 219 patients

The multicentre observational study analysed 661 assessments from 219 people with SCA27B to examine disease progression, clinical outcome metrics and demographic or genetic modifiers.

This multicentre, intercontinental observational study examined 661 assessments from 219 patients with spinocerebellar ataxia 27B (SCA27B), alongside 390 healthy controls, to investigate the Scale for the Assessment and Rating of Ataxia (SARA), longitudinal progression and potential modifiers of disease severity. Overall progression was 0.54 SARA points per year. Gait, stance and lower-limb impairment were the principal contributors to ataxia severity, while ageing-related motor variability and comorbidities affected assessment. The study also found that larger GAA repeat expansions on the shorter FGF14 allele were associated with faster progression.

Year of publication

2026

Source

eBioMedicine

Author

Andreas Traschütz, Ralf-Dieter Hilgers, Friedrich Erdlenbruch, Christel Depienne, Thomas Wirth, Clarisse Delvallée, Astrid Nümann, Catherine Ashton, David Pellerin, Elisabetta Indelicato, Felix Heindl, Mathilde Renaud, Max Borsche, Marcus Grobe-Einsler, Jennifer Faber, Thomas Klockgether, Ludger Schöls, Bernard Brais, Mathieu Anheim, Dagmar Timmann, Matthis Synofzik

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