News

People with Cushing’s syndrome who undergo surgery to remove one or both adrenal glands experience a persistent decrease in kidney function after one month that does not seem to worsen or improve with time, a study has found. The study, “Postoperative renal impairment and longitudinal change…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Making incisions through the back of the body, rather than the front, can lessen bleeding and shorten operation time during surgery to remove the adrenal glands, a new study suggests. The study, “Comparison of synchronous bilateral transperitoneal and posterior retroperitoneal laparoscopic adrenalectomy: results of a multicenter study,”…

More than half of the people with Cushing’s syndrome due to masses in both adrenal glands have disease-causing mutations the ARMC5 gene, a small study has found, suggesting that such patients should undergo genetic analysis for this gene. The study, “Allelic Variants of ARMC5 in Patients…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

While the diagnosis of Cushing’s syndrome is often based on a low-dose dexamethasone suppression test (DST), test results may be affected by differences in the metabolism of the steroid medication, a study found. The researchers advocated for routine measurement of dexamethasone levels during DST, saying test results should…

Isturisa (osilodrostat) is now approved in the U.S. for adults with Cushing’s disease for whom pituitary gland surgery is not an option or whose disease has returned after surgery, Recordati announced. The therapy is the first U.S. Food and Drug Administration (FDA)-approved medicine…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

Excess glucocorticoid levels in Cushing’s syndrome induces whole-body metabolic changes that are likely mediated by an inter-communication between different organs, a mouse study suggests. The study, “Correlation among body composition and metabolic regulation in a male mouse model of Cushing’s syndrome,” was published in the…

Recordati‘s U.S. marketing rights for Cushing’s disease medications Signifor (pasireotide) and Signifor LAR will be handled by the company’s Recordati Rare Diseases subsidiary. The medications will be marketed through Recordati Rare Diseases’ newly established endocrinology franchise, the company said. During the transfer…