RFC1 and cerebellar ataxia: The ARCA Registry has (i) enabled the harmonization of clinical outcomes across ataxia centers around the world; (ii) has demonstrated its capacity to act as a centralized database for genotype–phenotype and natural history studies in the >100 ARCAs, already exemplified for COQ8A-, RFC1-, and POLG-related ataxias; and (iii) aggregates the necessary large-scale longitudinal progression datasets for calculating sample sizes, modeling trial designs and randomization procedures, and running pharmacometric models simulating treatment effect sizes for anticipated clinical trials.