Measuring the levels of the RNA molecule that provides instructions to make a protein called FKBP5 could be useful for assessing cortisol activity in people with Cushing’s disease, a new study indicates. The study, “Evaluation of FKBP5 as a cortisol activity biomarker in patients with ACTH-dependent…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
The U.S. Patent and Trademark Office has issued a new patent to Strongbridge Biopharma that covers a method of using Recorlev (levoketoconazole), the company’s investigational treatment for endogenous Cushing’s syndrome, to treat people with Cushing’s who also are taking metformin for type 2 diabetes. According to a…
Some 25% of Cushing’s disease patients — or 1 in every 4 — will develop Nelson’s syndrome (NS) after surgery to remove both adrenal glands, while around 1 in every 5 will need pituitary-specific treatment, a study found. Lifelong follow-up is also needed for patients who undergo double adrenal gland…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
Symptoms of psoriasis vulgaris, an autoimmune disease that mainly affects the skin, eased in a man with cyclic Cushing’s disease during a short episode of hypercortisolemia, a period marked by cortisol levels rising above healthy limits, according to a case study.
Xeris Pharmaceuticals and Strongbridge Biopharma have entered an agreement in which Xeris will acquire Strongbridge, forming a new entity called Xeris Biopharma Holdings. Following the transaction, Xeris also will acquire Strongbridge’s pipeline, which includes Recorlev (levoketoconazole), an investigational treatment for endogenous Cushing’s syndrome. “Strongbridge’s attractive rare…
Sparrow Pharmaceuticals is planning to launch a Phase 2 clinical trial to assess the safety and effectiveness of its investigational, lead small molecule HSD-1 inhibitor, SPI-62, in people with Cushing’s syndrome. The trial is expected to open later this year, and it specifically will enroll people with ACTH-dependent Cushing’s…
Metopirone (metyrapone) safely and successfully maintained lower levels of urinary cortisol over nine months in adults with endogenous Cushing’s syndrome, according to data from the six-month extension of the PROMPT trial. Treatment with the HRA Pharma Rare Diseases…
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