News

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…

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…

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,…

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…