News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

To help improve the effectiveness of surgical treatment of Cushing’s disease, researchers conducted a study to determine common failures. They classified these failures into six different categories. Results were reported in the study, “Root cause analysis of diagnostic and surgical failures in the treatment of suspected Cushing’s…

Relacorilant, an investigational therapy developed by Corcept Therapeutics, may effectively manage the effects of excess cortisol in patients with Cushing’s syndrome, interim data from an ongoing Phase 2 trial show. In particular, the treatment significantly improved sugar tolerance and the levels of osteocalcin, a bone growth biomarker  commonly suppressed by…

Millendo Therapeutics‘ experimental small molecule ATR-101 shows promise as a treatment for Cushing’s disease in dogs, suggesting its potential in humans with the syndrome, according to a new study. It also suggests that dogs with naturally occurring Cushing’s could be used as a model for human disease. The disease is…

“Functional” adrenal incidentalomas — abnormal masses found by chance in the adrenal glands that are capable of secreting cortisol and causing Cushing’s syndrome — may occur more frequently than previously estimated, a small Japanese study suggests. These findings suggest that adrenal incidentalomas should be carefully examined, the researchers said.

While adrenocortical carcinoma — a malignant tumor of the adrenal gland — appears only rarely in children, the tumor may cause secondary Cushing’s syndrome in these patients, a new case report shows. Early diagnosis of the causes of Cushing’s syndrome could improve the prognosis of these children, researchers say. The…

In rare cases, Cushing’s syndrome may be caused by cortisol-secreting masses in both adrenal glands, a case report shows. The study with that finding, “ACTH-independent Cushing’s syndrome with bilateral cortisol-secreting adrenal adenomas: a case report and review of literatures” was published in BMC Endocrine Disorders. Cushing’s syndrome…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Obesity can be triggered by detrimental changes to the normal daily rhythms of hormone production, leading to an increased production of fat cells, research in mice shows. These findings help to explain why conditions that disturb hormone levels, such as Cushing’s disease…