Endocrine Abstracts

More than 90% of classic congenital adrenal hyperplasia (CAH) patients have defects in the cytochrome P450 enzyme steroid 21-hydroxylase, resulting in the inability to produce cortisol as well as the overproduction of androgens and androgen precursors such as 17-hydroxyprogesterone (17-OHP). Management of classic CAH can be challenging since patients are often unable to adequately balance the supraphysiologic doses of exogenous glucocorticoids required to suppress excess andro...

Background: Prader-Willi syndrome (PWS) is a rare disease characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity and early mortality and to a significant burden on patients and caregivers. There is no approved treatment for hyperphagia in PWS. Patients with PWS have increased circulating levels of the orexigenic hormone acylated ghrelin (AG) with a relative deficit of unacylated ghrelin (UAG). Livoletide (AZP-531) is a first-in-cla...

Background: Hot flushes (HF) affect 70% of menopausal women and can be debilitating. Oestrogen administration is effective but not without risk. Neurokinin B signalling is increased in menopausal women, and is likely critical in the aetiology of their HF. We therefore hypothesised that a neurokinin 3 receptor (NK3R) antagonist could attenuate menopausal flushing.Design: This single-centre, phase 2, randomised, double-blind, placebo-controlled, crossover ...

Background: Hot flushes (HF) affect 70% of menopausal women and can be debilitating. Oestrogen administration is effective but not without risk. Neurokinin B signalling is increased in menopausal women, and is likely critical in the aetiology of their HF. We therefore hypothesised that a neurokinin 3 receptor (NK3R) antagonist could attenuate menopausal flushing.Design: This single-centre, phase 2, randomised, double-blind, placebo-controlled, crossover ...