After receiving the approval of ketoconazole for the treatment of Cushing’s syndrome patients in the European Union, HRA Pharma is setting up to conduct a post-approval observation study that will closely monitor patients receiving the therapy for any adverse events. The study is part of the mandatory pharmacovigilance…
News
In some rare cases, Cushing’s disease patients have a benign pituitary tumor that produces several pituitary hormones in excess, rather than just the adrenocorticotropic hormone (ACTH). When these tumors also produce prolactin, they can be treated with dopamine agonists as an alternative to surgery, a case report suggests. The…
Removal of pituitary adenomas by inserting surgical instruments through the nose (transsphenoidal resection) remains the best treatment option for pediatric patients, despite its inherent technical difficulties, a new study shows. The study, “Transsphenoidal surgery for pituitary adenomas in pediatric patients: a multicentric retrospective study,” was…
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
Cushing’s syndrome should be suspected in children and adolescents with evident growth delays, and signs of rapid weight gain, depression, anxiety, and hyperandrogenism, a study finds. While brain surgery to remove a tumor lessens the symptoms of Cushing’s, many children and adolescents end up with…
The European Medicine Agency‘s Committee for Orphan Medicinal Products (COMP) has recommended that Corcept Therapeutics‘ relacorilant receive orphan drug designation for the treatment of Cushing’s syndrome, the company announced in a press release. The European Commission is expected to accept the recommendation by the end of the…
Mifepristone, a synthetic steroid commonly used to taper high blood sugar levels and diminish insulin resistance in patients with Cushing’s syndrome, may also be helpful in managing mild autonomous cortisol secretion (ACS) due to adrenal tumors, a Phase 4 pilot trial shows. Although ACS is considered a…
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…
In real-life clinical practice, Novartis‘ Signifor (pasireotide) completely or nearly normalizes the levels of cortisol in the urine of patients with mild to moderate Cushing’s disease, a study says. The study, “The medical treatment with pasireotide in Cushing’s disease: an Italian multicentre experience based on ‘real-world…
Strongbridge Biopharma is preparing the submission of a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) seeking the approval of Recorlev (levoketoconazole) for the treatment of patients with endogenous Cushing’s syndrome. According to recent statements from the company, the…
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