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Long-term study shows CRENESSITY improves growth in children with CAH

Two-year clinical data presented at ENDO 2026 indicate that children treated with CRENESSITY for classic congenital adrenal hyperplasia experience slowed bone age progression and improved predicted adult height, offering a potential shift in how clinicians manage the hormonal and physical complications of the rare genetic condition.

Long-term study shows CRENESSITY improves growth in children with CAH

The findings, drawn from the CAHtalyst Pediatric study, focus on a subset of growing patients who entered the trial with advanced bone age. Researchers observed that sustained androgen control, facilitated by the drug, allowed for meaningful reductions in glucocorticoid exposure. Among those with advanced skeletal maturity, the mean bone age standard deviation score decreased by 1.12 over two years, with some participants showing reductions exceeding two standard deviations. Consequently, mean predicted adult height in this group increased by 4.7 cm.

Maria G. Vogiatzi, principal investigator and division head of endocrinology at Children's Hospital of Philadelphia, noted that stabilizing bone age is a significant hurdle in pediatric care, as accelerated maturation often permanently limits a child's final height. Beyond clinical metrics, caregiver feedback highlighted high levels of treatment satisfaction. Every surveyed caregiver reported a willingness to recommend the therapy, while expressing increased optimism regarding their child’s future quality of life and a reduced reliance on high-dose steroids, which are often linked to metabolic risks like weight gain and insulin resistance.

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