Congenital Adrenal Hyperplasia (CAH) is a rare inherited disorder in which the adrenal glands do not function properly because of a variant in the gene for encoding adrenal steroid 21-hydroxylase, without this enzyme, the adrenal glands may produce too little cortisol and/or aldosterone and too much androgen.
Currently, there is no approved treatment specifically for CAH. The current goal of therapy, especially in adult patients, is to ensure proper hormone levels, reduce the burden of steroids in CAH management and improve overall quality of life.
Today, the only way doctors can help most people living with CAH is with high doses of steroids (glucocorticoids). This therapy can often lead to uncomfortable side effects, which is why it is important to have a team of healthcare providers, including an endocrinologist.
CAHmelia is a clinical trial researching an investigational medication for congenital adrenal hyperplasia. With your help, we can find a treatment specifically developed for CAH. By taking part in this clinical trial, you are leaving a mark on medicine and helping push new treatment options with widespread benefits. Clinical trials offer hope for many people and an opportunity to help researchers find better treatments for others in the future!
If you or a loved one have been diagnosed with classic CAH due to 21-hydroxylase deficiency, please visit CAHstudy.com today!