The US Food and Drug Administration (FDA) has declined to approve Applied Therapeutics’ govorestat for classic galactosemia, prolonging the wait for the first marketed treatment for the genetic ...

Background and aims: Hypoglycemia is a typical, potentially life-threatening symptom in classical galactosemia with blood galactose considerably elevated (up to ∼100 mg/dl). Therefore we tested ...

causes a buildup of galactose, leading to an improper conversion to the toxic metabolite galactitol. Accumulation of this molecule causes neurological complications, including deficiencies in ...