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December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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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 ... Read more

November 26, 2019November 26, 2019

EMA’s CHMP Recommends Approval of Isturisa as Treatment for Cushing’s Syndrome

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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 ... Read more

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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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. ... Read more

November 14, 2019

USP8 May Be Potential Target for Cushing’s Disease Therapy, Mouse Cell Study Suggests

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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 ... Read more

November 12, 2019

Symptoms Linked With Cardiovascular Risk More Prevalent in Subclinical Cushing’s, Review Finds

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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 ... Read more

November 7, 2019

Blood Clot Risk in Cushing’s Disease Patients May Extend Well Beyond Pituitary Surgery, Case Study Finds

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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, ... Read more

November 5, 2019

Genetic Variations of Glucocorticoid Receptor May Explain Loss of Muscle Strength in Cushing’s, Study Says

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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 ... Read more

October 31, 2019

Doctors Should Suspect Cushing’s Syndrome in Cases of Treatment-resistant Depression, Report Suggests

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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 ... Read more

October 29, 2019

Long-term Topical Corticosteroid Use can Cause Secondary Cushing’s Syndrome in Adults, Case Report Shows

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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, ... Read more

October 24, 2019

Poor Fat Metabolism and Insulin Resistance Linked to Bone Loss, Fracture Risk in Cushing’s Patients

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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. ... Read more

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