Lynch, D. R., Hauser, L. , McCormick, A. , Wells, M. , Dong, Y. N., McCormack, S. , Schadt, K. , Perlman, S. , Subramony, S. H., Mathews, K. D., Brocht, A. , Ball, J. , Perdok, R. , Grahn, A. , Vescio, T. , Sherman, J. W. and Farmer, J. M. (2019); Ann Clin Transl Neurol. doi:10.1002/acn3.731
Results
No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open‐label extension period, subjects had a more stable course than expected based on natural history data.
Conclusions
This study provides no direct evidence for a beneficial effect of IFN‐γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.
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