Crinecerfont Promising for Congenital Adrenal Hyperplasia

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New phase 3 data shows that Crinecerfont, an oral medication, is beneficial for both adults and children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. This genetic condition leads to impaired cortisol synthesis and excessive production of adrenal androgens. Current treatment involves high doses of glucocorticoids, which can have negative side effects. Crinecerfont reduces adrenal androgen production independently of glucocorticoids. Results from adult and pediatric trials showed a significant reduction in glucocorticoid doses with Crinecerfont therapy, leading to more physiologic hormone levels. The drug is seen as a promising new treatment option for CAH patients.

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