Neurologic outcomes in Friedreich ataxia

Massimo Pandolfo, Neurol Genet Jun 2020, 6 (3) e415; DOI: 10.1212/NXG.0000000000000415

The SARA is a sensitive outcome measure in ambulatory patients with FRDA and has an excellent correlation with functional capabilities. Ambulatory patients with onset before age 8 years showed the fastest measurable worsening. Loss of ambulation in high-risk patients is a disease milestone that should be considered as an end point in clinical trials.

Neurologic outcomes in Friedreich ataxia