More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
News
Clinical outcomes of COVID-19 in people with Cushing’s syndrome seem to depend on the severity of hypercortisolism, or how high blood cortisol levels are in these patients, according to a recent case series study. “Severe hypercortisolism is a warning sign that [Cushing’s syndrome] affected by COVID-19…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
Cortisol fluctuations in a woman with Cushing’s disease were controlled successfully with Signifor (pasireotide LAR), possibly for the first time, according to a recent case report. The report, “Resolution of cyclicity after pasireotide LAR in a patient with Cushing’s disease,” was published in the journal…
Recorlev (levoketoconazole), Strongbridge Biopharma’s oral investigational therapy for endogenous Cushing’s syndrome, led to sustained improvements in cortisol and in blood sugar control among patients with both Cushing’s and diabetes, a subgroup analysis of Phase 3 SONICS trial data showed. “Diabetes mellitus [type 1 diabetes] is among the…
Although mortality rates associated with endogenous Cushing’s syndrome have dropped in recent years, the disease is still linked to a threefold increase in the risk of death, mainly from cardiovascular issues and infections, a recent analysis of past research has found. “Our results found that death rates have fallen…
From bolstering a list of recommended physicians to sharing patient stories, the Cushing’s Support & Research Foundation (CSRF) offers various ways for supporters to mark Cushing’s Awareness Day, taking place on April 8. The day is set aside annually to call attention to the more than 6 million people…
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
The Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Isturisa (osilodrostat) to treat people with endogenous Cushing’s syndrome for whom pituitary brain surgery has not been effective or is not an option. Recordati…
Recorlev (levoketoconazole), Strongbridge Biopharma’s oral candidate to treat endogenous Cushing’s syndrome, was superior to a placebo at lowering cholesterol levels and body mass index, a measure of body fat, according to newly released data from the pivotal Phase 3 LOGICS trial. These findings, concerning most of the trial’s…
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