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

Patel, M. , Schadt, K. , McCormick, A. , Isaacs, C. , Dong, Y. N. and Lynch, D. R. (2019), Muscle & Nerve. Accepted Author Manuscript. doi:10.1002/mus.26610

Results
In comparisons of participants’ baseline and week 26 visits, only the pediatric cohort's 1MW score showed change (p<0 .05="" br="" change="" did="" measure="" not="" outcome="" primary="" significantly.="" t25fw="" the="">
Discussion
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.
Open label Pilot Study of Oral Methylprednisolone for the Treatment of Patients with Friedreich Ataxia