Predictors of loss of ambulation in Friedreich's ataxia

Christian Rummey, Jennifer M. Farmer, David R. Lynch; EClinicalMedicine, Volume 18, 2020, 100213, doi:10.1016/j.eclinm.2019.11.006.

We propose a stratification paradigm for time to LoA in FRDA. Concurrently, each step in a sequence of events represents a surrogate measure for future LoA. This will facilitate patient selection and stratification in clinical trials, and potentially enable study of LoA as a direct clinical outcome.

Predictors of loss of ambulation in Friedreich's ataxia