Marisa Wexler, MS,  senior science writer—

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…

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…

For people with Cushing’s disease whose pituitary tumor cannot be identified, removal of one-third of the pituitary gland followed by multidisciplinary treatment and care can lead to favorable outcomes, a small study suggests. The study, “Lateral one-third gland resection in Cushing patients with failed adenoma identification…

The U.S. Food and Drug Administration (FDA) is reviewing an application from Strongbridge Biopharma seeking the approval of Recorlev (levoketoconazole) for the treatment of endogenous Cushing’s syndrome. A final decision from the FDA is expected by Jan. 1, 2022 — meaning that Recorlev’s approval request will follow the…

Chronic insomnia can be a sign of Cushing’s syndrome, and doctors need be aware of this possibility, according to a case report of a woman diagnosed with Cushing’s shortly after seeking help for her insomnia. This case also emphasized the usefulness of the dexamethasone suppression test in identifying the specific…

HRA Pharma Rare Diseases is providing an unrestricted educational grant to fund “Cushing’s Hub,” a first-of-its-kind online platform designed to help healthcare providers care for patients with Cushing’s syndrome. The Hub, developed by Springer Healthcare Education, will provide information to promote good clinical practice, and improve diagnosis and…

Although rare, Cushing’s disease associated with a tumor in the pituitary gland — a pea-size gland found deep within the brain — may occur in patients who also have a meningioma, a type of tumor that arises from the membranes that cover the brain within the skull, a case…

The cancer medicine bexarotene may hold promise for treating Cushing’s disease, a study suggests. The study, “Targeting the TR4 nuclear receptor with antagonist bexarotene can suppress the proopiomelanocortin signalling in AtT‐20 cells,” was published in the Journal of Cellular and Molecular Medicine. Cushing’s disease is caused…