News

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

Recordati Rare Diseases announced that it has officially acquired marketing rights in Europe for the Cushing’s treatment Isturisa (osilodrostat), enabling the company to distribute it throughout the European Union, as well as in the U.K., Norway, Liechtenstein, and Iceland. The company — a subsidiary of Recordati, based in Italy…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

The pituitary adenomas that cause Cushing’s disease in men differ from those in women in hormone content and gene expression patterns, a study shows. These findings suggest that biological sex may also play a role in the molecular behavior of tumors, in addition to prevalence and clinical course. A…

As people with uncontrolled Cushing’s syndrome and adrenal insufficiency are at a relatively higher risk of infection, they are urged to follow Centers for Disease Control and Prevention guidance regarding COVID-19, and continue to use and ensure adequate supply of necessary medications. These were among recommendations issued…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

People with Cushing’s syndrome who undergo surgery to remove one or both adrenal glands experience a persistent decrease in kidney function after one month that does not seem to worsen or improve with time, a study has found. The study, “Postoperative renal impairment and longitudinal change…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…