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The clinical features and increased mortality associated with Cushing's syndrome result from a chronic excess of circulating cortisol. As LCI699 potently inhibits 11β-hydroxylase, which catalyzes the final step of cortisol synthesis, it is a potential new treatment for Cushing's disease, the most common cause of endogenous Cushing's syndrome.

the journal of clinical endocrinology and metabolism/journal of clinical endocrinology and metabolism

LCI699, a Potent 11β-hydroxylase Inhibitor, Normalizes Urinary Cortisol in Patients With Cushing's Disease: Results From a Multicenter, Proof-of-Concept Study