News

Strongbridge Biopharma‘s Recorlev (levoketoconazole) continues to show clinically meaningful improvements in the control of cortisol among patients with endogenous Cushing’s syndrome, a Phase 3 clinical trial shows. Cushing’s syndrome is caused by the excessive production of the hormone cortisol by the adrenal glands, which sit atop…

The annual incidence of Cushing’s disease in Sweden increased between 2005 and 2013 compared to earlier periods, according to nationwide data. However, this could be because of factors other than a truly higher disease frequency, including greater awareness and earlier diagnosis. Also, the findings demonstrate the importance of confirming Cushing’s…

Long-term treatment with Strongbridge Biopharma‘s Recorlev (levoketoconazole) continues to be well-tolerated and to induce clinically meaningful improvements in endogenous Cushing’s syndrome patients, an extended evaluation of a Phase 3 clinical trial shows. Cushing’s syndrome is a medical condition caused by the excessive production of the hormone cortisol by the…

Etomidate, a commonly used anaesthetic, is well-tolerated and can be used in low doses to reduce cortisol levels in patients with Cushing’s syndrome, a report of three cases suggests. The case series, “Low-dose etomidate for the management of severe hypercortisolaemia in different clinical scenarios: a case series and…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term…

Patients with high levels of cortisol but no clinical symptoms of Cushing’s syndrome can instead have a disease known as glucocorticoid resistance syndrome, according to a case report that documented a woman who had glucocorticoid resistance syndrome but without its associated genetic mutation. The study, “Cushing’s syndrome with no…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

In rare cases, the appearance of blood clots in arteries may be the first symptom of Cushing’s syndrome, a case report shows. That finding, “Arterial thrombosis as primary presentation of endogenous Cushing’s syndrome,” was published in the journal BMJ Case Reports. Cushing’s syndrome is a condition…

Stereotactic radiosurgery — a targeted form of radiation therapy — can effectively reduce hormone levels and control tumor progression in children and adolescents with hormone-secreting pituitary adenomas, particularly those with Cushing’s disease and acromegaly, a retrospective study shows. The study, “Outcomes after Gamma knife Stereotactic Radiosurgery in pediatric…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…