Larimar Therapeutics (LRMR) announced the publication of two peer-reviewed articles highlighting nonclinical data on the therapeutic potential, pharmacology, and mechanism of action of nomlabofusp as a novel frataxin protein replacement therapy designed to address the underlying cause of Friedreich’s ataxia, or FA. These data were included in the briefing package reviewed by the FDA in support of potentially using skin FXN concentrations as a reasonably likely surrogate endpoint for Larimar’s registrational program seeking accelerated approval for nomlabofusp.
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