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. 2022 Apr 11;18(6):337–352. doi: 10.1038/s41574-022-00655-w

Fig. 5. Novel therapeutic approaches in classic CAH.

Fig. 5

Novel treatment approaches include new ways to deliver circadian cortisol replacement (novel glucocorticoids) as well as various adjunct therapies to decrease adrenocortical androgen production, thereby enabling glucocorticoid dose reduction. These include direct adrenocortical steroidogenesis inhibitors and agents to suppress the hypothalamic–pituitary–adrenal axis (such as corticotropin-releasing factor (CRF) receptor 1 (CRF1) antagonists, an adrenocorticotropic hormone (ACTH)-specific monoclonal antibody and melanocortin type 2 receptor (MC2R) antagonists). Preclinical studies are exploring the role of restorative cell-based therapies. A first-in-human recombinant adeno-associated virus-based gene therapy in classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH) is also in development. Dashed arrows indicate processes blunted in CAH or targeted by abiraterone, a CYP17A1 inhibitor. Androgens and androgen precursors are highlighted in the deep blue boxes. Therapies are shown in yellow boxes. 17-OHP, 17-hydroxyprogesterone; DHEA, dehydroepiandrosterone; DHEA-S, DHEA sulfate.