Experimental drugs for Friedrich's ataxia: progress and setbacks in clinical trials

This editorial focuses on clinical trials in FA. A milestone in FA was set in 2023 on the emblematic day of rare diseases. On 28 February, the U.S. Food & Drug Administration approved omaveloxolone (Skyclarys®, Biogen Inc.) as the first FA specific therapeutic. Considering the numerous, negative, clinical trials concluded in FA, the exciting news of the omaveloxolone approval led the authors to reflect on the issues that made this step possible.

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