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The cortisol deficiency central to congenital adrenal hyperplasia requires lifelong treatment with distinct shifts in management required at each developmental stage.
A therapeutic strategy that reduces ACTH-driven androgen excess through a glucocorticoid-independent pathway could significantly improve treatment and patient QoL.
More than 6 in 10 adults treated with the investigational CRF1 antagonist crinecerfont reached a physiologic glucocorticoid dose with androstenedione control maintained.
Dr Richard Auchus says the ideal treatment for congenital adrenal hyperplasia is a long way off but describes a "block and replace" strategy that holds great promise.
Crinecerfont, a CRF1 antagonist, could help reduce glucocorticoid doses for individuals with CAH and improve physical and emotional quality of life, says leading researcher.
Richard J Auchus, MD, PhD, a preeminent steroid biologist, provides a concise and thorough primer on CAH and the challenges in managing the disorder safely and effectively.
Clinical guideline recommendations for optimal timing to gauge glucocorticoid treatment effects in congenital adrenal hyperplasia require individualization. Here's why.
In young adults with congenital adrenal hyperplasia of age to transition to adult care, 25% to 50% are not successful. Here are thoughts on problems and solutions.
Neurocrine Biosciences made the announcement and said, if approved in December, crinecerfont will be the first new treatment and treatment approach to CAH in 70 years.
The standard of care for congenital adrenal hyperplasia has not changed in more than 60 years nor have the challenges it presents been overcome.
Investigational treatments for congenital adrenal hyperplasia are focused on new ways to manage symptoms without the need for supraphysiologic doses of glucocorticoids.
Current therapies fall short of quelling the long-term exposure to and adverse effects of excessive androgen levels experienced by adults with CAH.
The registrational clinical trial met all endpoints related to androgen reduction and glucocorticoid dose reduction while maintaining androgen control.
Crinecerfont was granted FDA breakthrough therapy designation for the treatment of congenital adrenal hyperplasia.