CRN04894, a first-in-class, investigational oral medication to treat Cushing’s disease, demonstrated pharmacologic proof-of-concept with a dose-dependent suppression of cortisol, according to data from a Phase 1 study. CRN04894, developed by Crinetics Pharmaceuticals, is designed to block the interaction (antagonist) between the MC2R receptor, found specifically on the…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
Loss of muscle mass, also known as sarcopenia, is common among people with Cushing’s syndrome in long-term remission, and is associated with a poorer quality of life, a study reported. Its researchers also found the Sarcopenia Index, a blood test that can be used to estimate a patient’s muscle…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
A young woman with psychosis stemming from Cushing’s disease made a complete recovery through a combination of brain surgery and Metopirone (metyrapone) treatment, according to a case report. The researchers noted that Cushing’s disease is challenging to diagnose and often occurs alongside neuropsychiatric symptoms. Catching and treating…
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
A 62-year-old man with an aggressive form of Cushing’s disease had his diagnosis delayed because his symptoms initially resembled those of ectopic Cushing’s syndrome, according to a recent case report. Researchers said the case serves as an important reminder that “suspicion should be maintained” for a pituitary source…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
Blood tests should be performed before imaging examinations when Cushing’s syndrome is suspected, to prevent misdiagnosis, a recent case report study suggests. The report described the case of a 24-year-old woman with elevated cortisol levels and a pituitary tumor, in which an imaging test misled doctors into believing she had…
A 14-year-old girl with aggressive Cushing’s disease developed cavernous sinus thrombosis — a rare condition that causes blood clots to form in a cavity at the base of the brain — after an initial surgery to remove a benign tumor in the pituitary gland. Her case highlights that cavernous sinus…
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