Patricia Inácio, PhD, science writer —

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…

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…

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…

Isturisa (osilodrostat), an approved oral therapy for Cushing’s disease, can rapidly normalize — in a median of 35 days — the levels of urine free cortisol in people with the disease, according to newly released data from the Phase 3 LINC-4 trial. Treatment also led to sustained improvements in…

Strongbridge Biopharma has filed an application with the U.S. Food and Drug Administration (FDA) seeking the approval of Recorlev (levoketoconazole) to treat people with endogenous Cushing’s syndrome, the company announced. The application is supported by results from two Phase 3 studies, SONICS (NCT01838551) and LOGICS (NCT03277690), which showed…

Crinetics Pharmaceuticals is launching a Phase 1 clinical trial to evaluate the safety and tolerability of oral CRN04894, an inhibitor of adrenocorticotropic hormone (ACTH) activity, in healthy volunteers. The candidate therapy is being developed for Cushing’s disease and congenital adrenal hyperplasia (CAH), both characterized by abnormally high levels of…

Patients with active Cushing’s disease have lower blood levels of irisin, a hormone secreted by muscle cells, than those with controlled disease and healthy people, a study has found. Thus, measuring the levels of irisin may become a marker for diagnosing impairments of bone, muscle, and fat (adipose) tissues…

The first patient has been enrolled in Corcept Therapeutics‘ Phase 3 GRADIENT trial, which is evaluating relacorilant as a potential treatment for people with Cushing’s syndrome due to either an adrenal tumor or enlargement of the adrenal gland, the company announced. “GRADIENT is the first systematic…

Strongbridge Biopharma has finished enrolling patients in its Phase 3 LOGICS clinical trial assessing the safety and effectiveness of Recorlev (levoketoconazole) to treat endogenous Cushing’s syndrome, the company announced. “The completion of enrollment of the Phase 3 LOGICS trial for [Recorlev] represents a significant milestone for Strongbridge and…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…