Quantitative Gait and Balance Outcomes for Ataxia Trials: Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers

Type of Publication:
Ilg, Winfried
Milne, Sarah
Schmitz‑Hübsch, Tanja
Alcock, Lisa
Beichert, Lukas
Bertini, Enrico
Ibrahim, Norlinah Mohamed
Dawes, Helen
Gomez, Christopher M.
Hanagasi, Hasmet
Kinnunen, Kirsi M.
Minnerop, Martina
Németh, Andrea H.
Newman, Jane
Ng, Yi Shiau
Rentz, Clara
Samanci, Bedia
Shah, Vrutangkumar V.
Summa, Susanna
Vasco, Gessica
McNames, James
Horak, Fay B.
The Cerebellum
20 October 2023

With disease-modifying drugs on the horizon for degenerative ataxias, ecologically valid, fnely granulated, digital health measures are highly warranted to augment clinical and patient-reported outcome measures. Gait and balance disturbances most often present as the frst signs of degenerative cerebellar ataxia and are the most reported disabling features in disease progression. Thus, digital gait and balance measures constitute promising and relevant performance outcomes for clinical trials. This narrative review with embedded consensus will describe evidence for the sensitivity of digital gait and balance measures for evaluating ataxia severity and progression, propose a consensus protocol for establishing gait and balance metrics in natural history studies and clinical trials, and discuss relevant issues for their use as performance outcomes.