Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
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A European Medicines Agency (EMA) committee has recommended the approval of osilodrostat — to be marketed as Isturisa — for the treatment of adults with Cushing’s syndrome. Osilodrostat, formerly known as LCI699, is an oral treatment that inhibits an enzyme called 11-beta-hydroxylase, which is involved in…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
Targeting the USP8 protein, which is frequently mutated in Cushing’s disease patients, lowered the levels of adrenocorticotropic hormone, inhibited cell proliferation, and caused cell death in a mouse cell model of the disease, a study found. As a result, USP8 may be a promising target for therapies…
Symptoms associated with the risk of heart disease or stroke are common in people with asymptomatic tumors of the adrenal glands – called adrenal incidentalomas – particularly those that produce excess cortisol, a review of studies found. Problems such as high blood pressure, impaired blood…
People with Cushing’s disease are much more likely than others to develop blood clots, including rare ones in the brain, even after a pituitary surgery normalizes their cortisol levels, a case report shows. This finding suggests that patients should be managed with prophylactic (preventive) blood thinners from diagnosis and…
Genetic variations in the glucocorticoid receptor may explain why some patients with Cushing’s syndrome lose muscle strength, a study suggests. The study, “Glucocorticoid receptor polymorphisms influence muscle strength in Cushing’s syndrome,” was published in The Journal of Clinical Endocrinology & Metabolism. Cushing’s syndrome is caused…
Doctors should suspect Cushing’s syndrome in cases of depression that do not respond to medication or other interventions, avoiding misdiagnosis and unnecessary treatment, a case report suggests. While reducing cortisol levels often is enough to improve the depressive symptoms experienced by some Cushing’s patients — as occurred in this case…
Long-term use of topical corticosteroids to treat skin problems can trigger secondary Cushing’s syndrome in adults, with a severe impact on bone health, a case report shows. The study, “Osteoporotic vertebral fracture caused by topical corticosteroid abuse: A case report,” was published in the journal…
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Poor Fat Metabolism and Insulin Resistance Linked to Bone Loss, Fracture Risk in Cushing’s Patients
Abnormal fat accumulation, including increased bone marrow fat and insulin resistance, can lead to bone loss and a greater likelihood of fractures in people with Cushing’s disease, researchers report. Their study, “Beyond the…
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