Galactosemia (G) is due to either partial ... the level of Ts activity in erythrocytes with ovarian function. In 28 patients (pts), ages 1-32 yrs, Ts by microassay technique, LH, FSH and estradiol ...

Additional studies provided strong efficacy and safety data across 185 classic galactosemia patients over three years. Per Applied Therapeutics, approximately 3,300 patients in the United States ...

“During this time, we are committed to maintaining the expanded access programme for govorestat to ensure patients with Galactosemia have the opportunity to receive this important treatment.

the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data as well as positive interim trial results from the Phase III INSPIRE trial. Then, on December 2 ...

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 ...

the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data as well as positive interim trial results from the Phase III INSPIRE trial. Then, on December 2 ...