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…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
A man with exogenous, or therapy-related, Cushing’s syndrome, caused by long-term glucocorticoid use for Crohn’s disease, developed adrenal insufficiency after going into septic shock, a case study reported. This case highlights the dangers of adrenal insufficiency in patients on long-term glucocorticoid therapy during severe illness, and the need for quick…
People with Cushing’s disease who are treated with Isturisa (osilodrostat) should be monitored for the possibility of developing adrenal insufficiency, a report highlights. The case report, “Osilodrostat in Cushing’s disease: The risk of delayed adrenal insufficiency should be carefully monitored,” was published in the journal…
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