Open label Pilot Study of Oral Methylprednisolone for the Treatment of Patients with Friedreich Ataxia.

Pediatric participants improved in gait distance in the 1MW, but not significantly in other measures in this overall negative study. Methylprednisolone was generally well tolerated, suggesting that it may be useful for ambulatory children with FRDA if benefit is found with further study. This article is protected by copyright. All rights reserved.