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Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Dissolving hydrocortisone tablets in water for multiple daily dosing to treat congenital adrenal hyperplasia (CAH) in children may result in higher-than-intended dosing and exogenous Cushing’s syndrome — a form of the disease that occurs in those taking steroid hormones — a case report shows. The case highlights…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

Treatment with Isturisa (osilodrostat) led to a rapid and sustained reduction in cortisol levels and was well-tolerated in people with Cushing’s disease, according to newly published data from the LINC-3 clinical trial. The results were published in The Lancet Diabetes & Endocrinology, in the article “…

The first patient has been enrolled in Corcept Therapeutics‘ Phase 3 GRADIENT trial, which is evaluating relacorilant as a potential treatment for people with Cushing’s syndrome due to either an adrenal tumor or enlargement of the adrenal gland, the company announced. “GRADIENT is the first systematic…

Note: This story was updated Aug. 6, 2020, to note that ketoconazole is approved to treat Cushing’s in Europe and is used off-label for the same indication in the U.S. In cases where surgery is temporarily unavailable for Cushing’s syndrome patients due to the COVID-19 pandemic, a…

Isturisa (osilodrostat) is now available in Germany to treat adults with endogenous Cushing’s syndrome. The announcement by Recordati Rare Diseases, which acquired rights to Isturisa from its original developer Novartis, follows the medication recently becoming available in the U.S. and in France. Isturisa…

A newly developed smart contact lens might be used to measure levels of the hormone cortisol in a person’s tears, which could be helpful in disorders such as Cushing’s disease. The new device was described in Science Advances, in the study, “Smart, soft contact lens for…

Estrogen receptor alpha — one of the two main types of protein receptors activated by the female sex hormone estrogen — likely is involved in the development of Cushing’s syndrome during pregnancy. The study with that finding, “Estrogen receptor α plays an important role…