Galactosemia (G) is due to either partial or complete deficiency of the enzyme galactose-1-phosphate uridyl transferase (Ts). In females with G, there is a high incidence of premature ovarian ...

It also significantly reduced plasma galactitol levels in adults and children with galactosemia. Additional studies provided strong efficacy and safety data across 185 classic galactosemia ...

the Phase I/II ACTION-Galactosemia study in adults; as well as preclinical findings. If approved, govorestat would become the first medication indicated for the treatment of galactosemia and aid ...

Then, on December 2, 2024, Applied Therapeutics disclosed it received a "warning letter" from the FDA referring to the clinical trial issues underlying the CRL. Applied Therapeutics' common stock fell ...