New drug could help those with congenital adrenal hyperplasia reduce use of corticosteroids: NEJM
People born with the common form of congenital adrenal hyperplasia lack an enzyme in the adrenal glands needed to make the hormones cortisol and aldorsterone, which are involved in the body’s response to stress and blood pressure regulation.
The adrenals of children with this rare condition reroute hormone production to excess androgens, which tend to make them mature early and suffer from short stature and infertility as adults.
To make matters worse, a routine stomach virus can send CAH patients into what is known as an adrenal crisis, causing shock if not treated immediately.
The go-to therapy for children and adults with CAH are high dose corticosteroids, which come with a host of side effects, including weight gain, diabetes, osteoporosis and cognitive dysfunction.
A study, led by Richard Auchus, M.D., Ph.D., of the U-M Medical School Department of Pharmacology and internal medicine and investigators from the CAHtalyst Adult Trial provides strong evidence for an effective compound, crinecerfont, that could allow people with CAH to reduce their dose of replacement corticosteroids.
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