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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…

Corcept Therapeutics is recruiting participants for its Phase 3 clinical trial evaluating relacorilant as a potential treatment for Cushing’s syndrome-related side effects such as high blood pressure and impaired glucose tolerance. Also, findings from the study “A Randomized-Withdrawal, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and…

Serum cortisol and adrenocorticotropic hormone (ACTH) levels may be useful in identifying Cushing’s syndrome patients at risk of experiencing low blood potassium levels while being treated with Korlym (mifepristone), Corcept Therapeutics’ researchers report. Their work, “Anticipating Hypokalemia in Patients With ACTH-Dependent Cushing Syndrome Treated…

Elevated cortisol levels in people with subclinical Cushing’s syndrome appear to increase their likelihood of metabolic disease — a group of factors that raise the risk of heart disease — even though these patients don’t have the typical weight gain seen in overt Cushing’s syndrome, a study suggests. Results of the…