CAH Is Entering a New Era: Commercial Execution Will Determine the Winner

Executive Summary Congenital adrenal hyperplasia (CAH) remains one of the most challenging endocrine disorders to manage despite decades of therapeutic advances. Current treatment relies primarily on lifelong glucocorticoid replacement, often administered at supraphysiologic doses to suppress excessive adrenocorticotropic hormone (ACTH) secretion and adrenal androgen production. While effective in preventing adrenal crisis, chronic glucocorticoid therapy is associated with obesity, osteoporosis, diabetes, cardiovascular disease, impaired growth, infertility, reduced quality of life, and…