News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

The oral investigational treatment Recorlev (levoketoconazole) leads to sustained improvements in the control of cortisol among patients with endogenous Cushing’s syndrome, a Phase 3 trial shows. The trial’s findings were reported in the study “Efficacy and safety of levoketoconazole in the treatment of endogenous Cushing’s…

In patients with Cushing’s disease, removing the pituitary tumor via an endoscopic transsphenoidal surgery (TSS) leads to better remission rates than microscopic TSS, according to new research. But regardless of surgical approach, plasma cortisol levels one day after surgery are predictive of remission, researchers found. The study, “…

Delusions — fixed beliefs that do not change even when confronted with refutable evidence — are among the mental manifestations that can be  primary symptoms of Cushing’s disease, a case report suggests. The study, “Losing my mind: an uncommon presentation of Cushing’s disease,” was published in…

Data from an ongoing real-world study show that treatment with Signifor (pasireotide) is able to normalize cortisol levels for sustained periods in patients with Cushing’s disease for whom surgery has failed or is not an option. Half of the new Signifor users reached normal cortisol…

Adrenal gland transplants resulted in complete resolution of Cushing’s disease symptoms after no long-lasting benefits were seen with pituitary surgery, according to a new long-term study in four patients. The procedure also enabled lower doses of hormone replacement therapy, the findings showed. The research, “Autologous Adrenal…

People living with HIV who develop Cushing’s syndrome due to drug interactions tend to have more severe symptoms than non-infected people, a study found. The study, “Cushing’s syndrome due to interaction between ritonavir or cobicistat and corticosteroids: a case-control study in the French Pharmacovigilance Database,”…

Fluconazole — a treatment for fungal infections that can lower cortisol levels — could be a potential therapy for severe Cushing’s syndrome caused by ACTH-secreting tumors located outside the pituitary gland, a case report suggests. According to the report, “Fluconazole treatment in severe…

The administration with food of the Cushing’s syndrome therapy Korlym (mifepristone) will be covered by a new U.S. patent, according to Corcept Therapeutics – the company marketing Korlym. Upon issuance by the U.S. Patent and Trademark Office, Corcept plans to list the patent in the U.S.

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…