More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
Articles by Patricia Inácio, PhD
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Recorlev (levoketoconazole), Strongbridge Biopharma’s oral candidate to treat endogenous Cushing’s syndrome, was superior to a placebo at lowering…
Isturisa (osilodrostat), an approved oral therapy for Cushing’s disease, can rapidly normalize — in a median of 35 days…
Strongbridge Biopharma has filed an application with the U.S. Food and Drug Administration (FDA) seeking the approval of Recorlev (levoketoconazole) to treat…
Crinetics Pharmaceuticals is launching a Phase 1 clinical trial to evaluate the safety and tolerability of oral CRN04894, an…
Patients with active Cushing’s disease have lower blood levels of irisin, a hormone secreted by muscle cells, than those…
The first patient has been enrolled in Corcept Therapeutics‘ Phase 3 GRADIENT trial, which is evaluating relacorilant…
Strongbridge Biopharma has finished enrolling patients in its Phase 3 LOGICS clinical trial assessing the safety and effectiveness of…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…