News

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

The oral chemotherapy temozolomide might be an effective treatment for Cushing’s disease caused by aggressive tumors in the pituitary gland that continue to grow after surgery and taking other medications, a case report suggests. The study, “Successful reduction of ACTH secretion in a case of…

Clinical outcomes of Cushing’s disease patients who undergo endoscopic endonasal surgery to remove a pituitary adenoma are similar to those seen in patients who underwent previously used microscope-assisted techniques. According to a new study, that means despite the need for specialized training and equipment, using an endoscopic endonasal approach…

Giving corticotropin-releasing hormone (CRH) to Cushing’s disease patients helps improve the detection of pituitary tumors on positron emission tomography (PET) scans, increasing the chances of a more successful surgery, a study shows. The study, “CRH stimulation improves 18F-FDG-PET detection of pituitary adenomas in Cushing’s disease,” was published in the journal…

After receiving the approval of ketoconazole for the treatment of Cushing’s syndrome patients in the European Union, HRA Pharma is setting up to conduct a post-approval observation study that will closely monitor patients receiving the therapy for any adverse events. The study is part of the mandatory pharmacovigilance…

In some rare cases, Cushing’s disease patients have a benign pituitary tumor that produces several pituitary hormones in excess, rather than just the adrenocorticotropic hormone (ACTH). When these tumors also produce prolactin, they can be treated with dopamine agonists as an alternative to surgery, a case report suggests. The…

Removal of pituitary adenomas by inserting surgical instruments through the nose (transsphenoidal resection) remains the best treatment option for pediatric patients, despite its inherent technical difficulties, a new study shows. The study, “Transsphenoidal surgery for pituitary adenomas in pediatric patients: a multicentric retrospective study,” was…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Cushing’s syndrome should be suspected in children and adolescents with evident growth delays, and signs of rapid weight gain, depression, anxiety, and hyperandrogenism, a study finds. While brain surgery to remove a tumor lessens the symptoms of Cushing’s, many children and adolescents end up with…

The European Medicine Agency‘s Committee for Orphan Medicinal Products (COMP) has recommended that Corcept Therapeutics‘ relacorilant receive orphan drug designation for the treatment of Cushing’s syndrome, the company announced in a press release. The European Commission is expected to accept the recommendation by the end of the…