News

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Rare Case of Cushing’s Due to Tumors on Both Adrenal Glands

A 43-year-old woman developed Cushing’s syndrome due to bilateral adrenal adenomas, or a tumor on each of her two adrenal glands, according to a recent case report. “Although Cushing’s syndrome due to bilateral adrenal adenoma is rare, it should be considered in mid-aged females with typical symptoms,” researchers wrote. The…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Post-surgery Use of Enoxaparin Lowered Blood Clot Risk

Treatment with the blood thinner enoxaparin within a few hours after curative pituitary surgery may help reduce the risk of blood clots forming in people with Cushing’s disease, a new study suggests. The study “Venous thromboembolic (VTE) prophylaxis in Cushing disease patients undergoing transsphenoidal surgery” was published in…