News

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Although rare, pituicytomas should still be considered as a possible cause of Cushing’s disease in patients experiencing the typical symptoms of the disease, a case report highlights. The study, “Pituicytoma associated with Cushing’s disease: a case report and literature review,” was published in the…

HRA Pharma Rare Diseases has named AllianceRx Walgreens Prime as its exclusive pharmacy distributor for two rare disease medications: Metopirone (metyrapone), used to diagnose Cushing’s syndrome, and Lysodren (mitotane), an approved treatment for people with adrenocortical carcinoma, a rare cancer of the adrenal…

Two cases of Cushing’s syndrome that were diagnosed following pregnancy are described in a new report, which highlights the difficulty of recognizing the rare disorder in pregnant women. The report, “Cushing’s syndrome in pregnancy, diagnosed after delivery,” was published in Yeungnam University Journal of Medicine in…

Isturisa (osilodrostat) rapidly and safely controlled high cortisol levels associated with severe Cushing’s syndrome in three cancer patients who could not take standard medications, a case series study reported. The study, “Efficacy and tolerance of osilodrostat in patients with severe Cushing’s syndrome due to…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…