News

MicroRNA Signature Linked to Rare Cause of Cushing’s

There are sets of microRNAs that may be used as markers for inherited or newly arising primary macronodular adrenal hyperplasia (PMAH), a rare cause of Cushing’s syndrome, a small study found. According to researchers, these microRNAs — short RNA molecules that help control the activity of certain genes — also…

Q&A With RARE-X Disease Data Platform Founder, Nicole Boice

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

Corcept Will Seek FDA Approval of Relacorilant in 2023

Corcept Therapeutics is on track to ask the U.S. Food and Drug Administration (FDA) to approve the investigational therapy relacorilant to treat Cushing’s syndrome by the middle of next year, the company announced. Cushing’s syndrome is characterized by excessive levels of the stress hormone cortisol, which causes a range of…

Rare Disease Day Events Bring Awareness, Equity to Patients

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Cushing’s Relapse After Surgery May Be Sign of Cancer Spread

When symptoms of Cushing’s syndrome return after adrenal cancer surgery, this may indicate that tumor cells have spread to other locations, a case report suggests. The report, “Functioning metastasis from adrenocortical carcinoma,” was published in the journal BMJ Case Reports. Malignant tumors in the adrenal glands —…

High Blood Sugar Levels Reduced in Most Cushing’s Procedures

Hyperglycemia, or high concentrations of sugar in the blood, can be brought to normal levels, or considerably reduced, in almost 70% of people with both Cushing’s syndrome and hyperglycemia following curative surgery or radiation therapy, a study has found. The report highlighted the need for close monitoring and adjusting medications…

Xeris’ Recorlev Now Available in US From PANTHERx Rare Pharmacy

Xeris Biopharma’s newly approved therapy Recorlev (levoketoconazole) is now available to patients in the U.S. through the specialty pharmacy PANTHERx Rare, the company announced in a press release. The therapy is prescribed for adults with endogenous Cushing’s syndrome for whom surgery is not possible or not effective. “The commercial…