AT-007 is primarily used in the treatment of galactosemia, a rare genetic disorder that affects the body's ability to process galactose. It works by inhibiting the production of toxic metabolites that accumulate due to the enzyme deficiency in this condition. By reducing these harmful substances, AT-007 helps alleviate symptoms and prevent long-term complications such as liver damage, cognitive impairment, and cataracts. Additionally, it is being studied for its potential therapeutic effects in other metabolic disorders where similar pathways are involved. Its ability to modulate enzyme activity makes it a promising candidate for addressing a range of related conditions.