Nonclinical findings provide evidence of the mechanism of action of nomlabofusp and support the potential use of skin FXN concentrations as a novel surrogate endpoint for Larimar’s planned BLA ...

Under the new leadership of Marty Makary, the FDA should focus more attention on the critical role of real-world clinical data — such as from health insurance databases — in the ongoing assessment of ...

- NDA assigned a Target Action Date of February 25, 2026 - ...