News

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

SteroTherapeutics and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) are teaming up to conduct a Phase 2 clinical trial to determine the safety, efficacy, and dosage regimen for fluasterone as a potential treatment for the metabolic manifestations of Cushing’s syndrome. “This…

The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life is like with a rare disease, while raising awareness through their work of these disorders. Submissions for next year’s awards are open until Jan. 31. The competition is organized by France-based EURODIS, an alliance…

Recorlev (levoketoconazole), Strongbridge Biopharma’s oral investigational therapy, lessens disease symptoms and depression, and improves quality of life among people with endogenous Cushing’s syndrome, according to data from the Phase 3 SONICS trial. Notably, the therapy was found to particularly ease symptoms among female patients. “People with Cushing’s syndrome…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

People undergoing regular epidural steroid injections (ESIs) to ease back pain may be at increased risk of adrenal insufficiency, a preliminary study suggests. However, the researchers found no link between ESIs and Cushing’s syndrome. The study, “Incidence of Adrenal Insufficiency and Cushing’s Syndrome After Long-Term…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Researchers report a very rare case of Cushing’s disease in a 34-year-old woman caused by a “collision” brain tumor, a tumor simultaneously consisting of a meningioma and a pituitary adenoma. Meningioma, the most common type of brain tumor, arises from the meninges — the protective layers of tissue around…

Patients with ectopic Cushing’s syndrome face challenges in their healthcare management if they live in developing countries, according to a study of cases in Colombia that describes challenges unique to the tropics. “This study may lead to the formation of groups of excellence for the management of [Cushing’s…

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…