The company revealed encouraging news from the U.S. Food and Drug Administration (FDA), which has expressed openness to consider skin frataxin (FXN) concentrations as a reasonably likely surrogate ...

Nomlabofusp is a protein replacement therapy aimed at addressing the root cause of FA by delivering frataxin to mitochondria. The FDA’s decision to include it in the START program was based on ...

Lexeo Therapeutics announced positive interim data for LX2006 in Phase 1/2 studies targeting Friedreich ataxia (FA) cardiomyopathy, indicating surpassing results in frataxin expression and left ...

frataxin expression to be evaluated for any increase from baseline rather than numerical threshold Interim update from cohort 1 of LX2020 HEROIC-PKP2 Phase 1/2 trial: observed 71% and 115% ...

Participants have experienced clinically meaningful improvements across multiple measures, as well as increased frataxin expression in the heart, all of which underscore the potential of LX2006 to ...