News

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

Long-term treatment with Signifor LAR, a long-acting formulation of pasireotide, effectively reduced pituitary tumor volume and improved symptoms of Cushing’s disease, results from a Phase 3 extension trial show. These findings were discussed in the poster, “Long-Term Efficacy and Safety of Once-Monthly Pasireotide in Patients…

Radiation therapy targeting the entire sella — the compartment where the pituitary gland resides at the base of the brain — is effective at controlling Cushing’s disease and should be considered for patients with suspected invasive adenoma tumors, a new study shows. The study, “Technique of Whole-Sellar Stereotactic Radiosurgery for…

Cushing’s disease patients with macroadenomas — pituitary tumors larger than 10 mm — should undergo transsphenoidal pituitary surgery using the endoscopic technique, according to a new systematic review. The study, “Endoscopic vs. microscopic transsphenoidal surgery for Cushing’s disease: a systematic review and meta-analysis,” was published in…

Heart problems in Cushing’s syndrome patients may be partially reversed after tumor removal surgery, new research shows. For most patients in the study, the surgery also improved quality of life, mental and physical health, anxiety, and depression. However, 1 in 3 patients showed worse anxiety and depression scores, researchers…

Low levels of some hormone receptors in patients with subclinical Cushing’s syndrome and in those with a type of adrenal gland tumor called pheochromocytoma, all with abnormal hormone levels in their blood, were linked to weight gain and obesity, a risk factor for cardiovascular and other diseases. Increases in…

Etomidate — a steroid synthesis blocker — is an effective treatment for patients with severe Cushing’s syndrome who do not respond to ketoconazole, according to a new case report from Mexico. The report, “Etomidate in the control of severe Cushing’s syndrome by neuroendocrine carcinoma,” appeared…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

To help improve the effectiveness of surgical treatment of Cushing’s disease, researchers conducted a study to determine common failures. They classified these failures into six different categories. Results were reported in the study, “Root cause analysis of diagnostic and surgical failures in the treatment of suspected Cushing’s…