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Cushing’s syndrome should be suspected in children and adolescents with evident growth delays, and signs of rapid weight gain, depression, anxiety, and hyperandrogenism, a study finds. While brain surgery to remove a tumor lessens the symptoms of Cushing’s, many children and adolescents end up with…

The European Medicine Agency‘s Committee for Orphan Medicinal Products (COMP) has recommended that Corcept Therapeutics‘ relacorilant receive orphan drug designation for the treatment of Cushing’s syndrome, the company announced in a press release. The European Commission is expected to accept the recommendation by the end of the…

Mifepristone, a synthetic steroid commonly used to taper high blood sugar levels and diminish insulin resistance in patients with Cushing’s syndrome, may also be helpful in managing mild autonomous cortisol secretion (ACS) due to adrenal tumors, a Phase 4 pilot trial shows. Although ACS is considered a…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

In real-life clinical practice, Novartis‘ Signifor (pasireotide) completely or nearly normalizes the levels of cortisol in the urine of patients with mild to moderate Cushing’s disease, a study says. The study, “The medical treatment with pasireotide in Cushing’s disease: an Italian multicentre experience based on ‘real-world…

Strongbridge Biopharma is preparing the submission of a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) seeking the approval of Recorlev (levoketoconazole) for the treatment of patients with endogenous Cushing’s syndrome. According to recent statements from the company, the…

Metoclopramide, a gastrointestinal medicine, can increase cortisol levels after unilateral adrenalectomy — the surgical removal of one adrenal gland — and conceal adrenal insufficiency in bilateral macronodular adrenal hyperplasia (BMAH) patients, a case report suggests. The study, “Retention of aberrant cortisol secretion in a patient with…

Strongbridge Biopharma’s Recorlev (levoketoconazole) alleviates clinical signs and symptoms of Cushing’s syndrome that may cause discomfort and distress to patients, a Phase 3 trial shows. The trial’s findings were presented in a poster titled, “Levoketoconazole in the Treatment of Endogenous Cushing’s Syndrome: Improvements…

Mifepristone, a medicine that blocks glucocorticoid hormone effects, may be useful for preventing adrenal insufficiency in people with Cushing’s syndrome, following surgery to remove adrenal glands, a study using rats suggests. The study, “Glucocorticoid Receptor Antagonist Administration Prevents Adrenal…

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…