May also be called: Galactose-1-Phosphate Uridyltransferase Deficiency; Galactokinase Deficiency; Galactose-6-Phosphatase Emirase Deficiency; GALT Deficiency; Galactose Epimerase Deficiency; Classic ...
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Applied Therapeutics' Govorestat Faces 'Significant' Uncertainty in Galactosemia Treatment After FDA Rejection, RBC Says"Govorestat has the potential to change the lives of patients with galactosemia, which we believe is evidenced by the breadth of efficacy and safety data demonstrating its ability to stop the ...
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 ...
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 ...
Metabolic disorders were the cause of ALF in 9 patients (43.47%): 3 infants with mitochondrial disorder, 2 infants with galactosemia and tyrosinemia respectively, and one with neonatal ...
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 ...
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