News

Although rare, Cushing’s disease associated with a tumor in the pituitary gland — a pea-size gland found deep within the brain — may occur in patients who also have a meningioma, a type of tumor that arises from the membranes that cover the brain within the skull, a case…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

Strongbridge Biopharma has filed an application with the U.S. Food and Drug Administration (FDA) seeking the approval of Recorlev (levoketoconazole) to treat people with endogenous Cushing’s syndrome, the company announced. The application is supported by results from two Phase 3 studies, SONICS (NCT01838551) and LOGICS (NCT03277690), which showed…

Disruptions in blood sugar metabolism and hypercortisolism — excessive levels of the hormone cortisol in the blood — are both linked to a higher risk of hyperlipidemia, or having high levels of fatty molecules circulating in the bloodstream, in people with Cushing’s disease, a study found. Hyperlipidemia, in turn,…

For years before and after their diagnosis, people with Cushing’s disease use more psychotropic medications — those that affect mood, thoughts, or perception — for mental health problems than their healthy peers, a study in Sweden found. Notably, patients experiencing long-term disease remission still showed higher use of antidepressants…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Crinetics Pharmaceuticals is launching a Phase 1 clinical trial to evaluate the safety and tolerability of oral CRN04894, an inhibitor of adrenocorticotropic hormone (ACTH) activity, in healthy volunteers. The candidate therapy is being developed for Cushing’s disease and congenital adrenal hyperplasia (CAH), both characterized by abnormally high levels of…