Metopirone (metyrapone) safely normalized, or at least lowered by half, the levels of cortisol found in the urine of patients with endogenous Cushing’s syndrome, according to data from a Phase 3/4 clinical trial. Findings from the study, called PROMPT (NCT02297945), were presented recently by…
News
A combination of bexarotene and lapatinib, two approved anti-cancer medications, may hold promise in treating Cushing’s disease, according to a recent study. The study, “Bexarotene combined with lapatinib for the treatment of Cushing’s disease: evidence based on drug repositioning and experimental confirmation,” was published in the…
Patients with endogenous Cushing’s syndrome who stopped using Recorlev (levoketoconazole) and moved to a placebo in a study started having their urine cortisol levels rise in response to lack of treatment, compared with those who remained on Recorlev, according to top-line data from the Phase 3 LOGICS…
Abnormally high levels of cortisol in the urine — one of the hallmarks of Cushing’s syndrome — seem to be associated with alterations in blood sugar metabolism in obese patients, a study found. The study, “Hypercortisolism and altered glucose homeostasis in obese patients in…
An alternative diagnostic test to measure the levels of late-night cortisol in the saliva of patients with Cushing’s disease did not meet the standards of currently used methods, a study demonstrates. The study, “Prospective evaluation of…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
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…
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