Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). The enzyme responsible for production of the toxic metabolite, galactitol, is aldose reductase. This medicine, also known as AT-007, blocks the activity if aldose reductase, preventing conversion of galactose to toxic galactitol.

Based on description provided by sponsor