There is an urgent need for a non-steroidal treatment in children diagnosed with classic CAH to enable more effective management of the disease early in life.

Because of the challenges of the disease, most countries in the developed world have active screening programs in newborns to detect classic CAH. When diagnosed, children with classic CAH are immediately started on glucocorticoid (steroid) therapy to replace the missing cortisol that is characteristic of this disease, and will need to stay on this therapy for life.  In addition, approximately 70% of classic CAH patients have the salt-wasting form of the disease, and are also started on mineralocorticoid therapy.

In children, a secondary feature of classic CAH, specifically overproduction of male sex hormones such as androstenedione and testosterone, can result in premature puberty appearing as early as five years of age. At the same time, the treatment for classic CAH, glucocorticoids, affects bone development and can negatively impact a child’s growth trajectory. Children with classic CAH are in urgent need of a non-steroidal treatment to enable control of male sex hormones to prevent early puberty and other health effects, while also allowing reduced steroid usage to minimize impact on bone development.

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