Neurological decline in people with Friedreich ataxia

Takeaway

  • The Scale for the Assessment and Rating of Ataxia (SARA) strongly correlates with neurological function and is an effective outcome measure in ambulatory individuals with Friedreich ataxia (FRDA).

  • Ambulatory loss represents a disease milestone in high-risk individuals.

Why this matters

    Improving knowledge of the neurological disease course will help to improve the design of clinical trials, at a time when novel therapeutics are likely to enter the clinical field.