Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
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Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. 20 webinar. William Whitman…
Mutations in Internal Clock Regulators May Be Root Cause of Cyclic Cushing’s, Case Report Suggests
Development of cyclic Cushing’s disease — a form of Cushing’s in which cortisol levels are intermittently high — may be a result of mutations in genes controlling both pituitary tumor growth and the body’s internal clock system, a case report suggests. The study, “A putative…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
Cushing’s syndrome can occur during pregnancy without obvious signs and symptoms specific to the condition, a new case report shows, suggesting the disease should be considered as a diagnosis in cases of high blood pressure during early pregnancy. The case report, “A case of subclinical Cushing’s…
A rare case of a woman with both endogenous and exogenous Cushing’s syndrome, demonstrating that both forms of the disease can coexist, is described in a case report. The case study, “A Curious Case of ‘Double Cushing Syndrome,’” was published in the journal AACE Clinical…
In addition to changes in volume of certain brain structures, people with Cushing’s syndrome also show widespread changes in the consistency of brain tissue, namely in white matter, which is made of nerve fibers or axons. Importantly, such changes may correlate with disease symptoms. The study with those findings,…
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
Secretion of multiple hormones, high morning cortisol levels, higher tumor grade, and diabetes are all significantly associated with a worse prognosis for people with ectopic Cushing’s syndrome (ECS) caused by a neuroendocrine tumor, a new study found. The study, “Multiple hormone secretion may indicate…
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