Strongbridge Biopharma released additional positive results from a Phase 3 trial evaluating whether the company’s investigational therapy Recorlev (levoketoconazole) is safe and effective for people with endogenous Cushing’s syndrome. The latest results were presented in the scientific poster “Safety and Efficacy of Levoketoconazole in Cushing Syndrome: …
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Pituitary tumor conditions, such as Cushing’s disease, have a substantial effect on patients’ work capabilities and health-related quality of life, researchers from The Netherlands reported. The study, “Work disability and its determinants in patients with pituitary tumor-related disease,” was published in the journal Pituitary. Pituitary tumors, like…
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…
A recent case report describes a 7-year-old boy with Cushing’s disease who had an unusual clinical presentation, which significantly delayed his diagnosis. The study, “A variable course of Cushing’s disease in a 7 year old: diagnostic dilemma,” was published in the Journal of Pediatric Endocrinology and Metabolism.
Cushing’s disease patients whose pituitary tumors carry a USP8 mutation are more likely to achieve remission after surgery than those without such mutations, a retrospective Italian study found. The study, “Clinical characteristics and surgical outcome in USP8-mutated human adrenocorticotropic hormone-secreting pituitary adenomas,” was published in the journal…
An important cellular pathway involved in multiple cancers could potentially be targeted to treat adrenal cancers that cause Cushing’s syndrome, a study has found. The study, “MAPK/ERK pathway inhibition is a promising treatment target for adrenocortical tumors,”…
A patient with depression developed Cushing’s syndrome (CS) because of a rare ACTH-secreting small cell cancer of the prostate, a case study reports. The case report, “An unusual cause of depression in an older man: Cushing’s syndrome resulting from metastatic small cell cancer of the…
Keytruda Found Ineffective in Rare Case of Adrenal Cancer Caused by Lynch Syndrome, Report Shows
Keytruda (pembrolizumab) may not be effective in Lynch syndrome patients — those with inherited mutations in certain DNA repair genes — who develop Cushing’s syndrome due to cortisol-secreting metastatic adrenal cancer, a case report suggests. The report, “Rapid disease progression in a patient with mismatch…
Most Subclinical Cushing’s Patients Don’t Require Glucocorticoids After Adrenalectomy, Study Finds
Patients with subclinical hypercortisolism, i.e., without symptoms of cortisol overproduction, and adrenal incidentalomas recover their hypothalamic-pituitary-adrenal (HPA) axis function after surgery faster than those with Cushing’s syndrome (CS), according to a study. Moreover, the researchers found that an HPA function analysis conducted immediately after the surgical removal of adrenal…
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…
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