News

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

The Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency (EMA), is recommending that Alexion Pharmaceuticals‘ Ultomiris (ravulizumab-cwvz) be approved to treat children and adults with atypical hemolytic uremic syndrome (aHUS). A final decision from the European Commission is expected in the…

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…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

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…

Treatment with Soliris (eculizumab) during pregnancy may help prevent subsequent relapses of pregnancy-associated atypical hemolytic uremic syndrome (aHUS), according to a case report. The report, “Eculizumab Maintenance and the Prevention of Atypical Hemolytic Uremic Syndrome Relapse During Pregnancy: A Case Report,” was published in the…

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),…

A medication for high blood pressure, Tekturna (aliskiren) may be used as an add-on therapy to manage hypertension associated with atypical hemolytic uremic syndrome (aHUS) until more adequate treatments become available, a case report finds. The study, “Aliskiren as an adjunct therapy for atypical hemolytic uremic…

Atypical hemolytic-uremic syndrome can arise following an aortic dissection — a serious condition in which the inner layer of the aorta tears open — according to a new case report. The study, “Atypical Hemolytic Uremic Syndrome following Acute Type A Aortic Dissection,” was published in the journal…