Increased endogenous cortisol production was achieved in all patients at higher doses. A maximum change from baseline post-ACTH stimulation test of 4.7 µg/dL and 6.6 µg/dL was observed at the two ...
Inborn errors of steroidogenesis associated with congenital adrenal hyperplasia are discussed, with a particular focus on the pathophysiology and clinical features of 21-hydroxylase deficiency. The ...
BridgeBio Pharma, Inc. has released the topline results from the phase 1/2 open-label adventure study investigating BBP-631, an investigational gene therapy for congenital adrenal hyperplasia (CAH).