News

Erythropoietin (EPO) treatment safely promotes sustained increases in hemoglobin levels in people with cold agglutinin disease (CAD) and other autoimmune hemolytic anemias (AIHAs), according to a multicenter study in Europe. The data pointed to EPO as a safe and effective treatment for CAD, especially if given within the first…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

A 46-year-old woman who tested positive for the new coronavirus also was diagnosed with cold agglutinin disease (CAD), a case study reported.  The link between the COVID-19-causing virus and CAD needs to be monitored, scientists said. The study, “…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

The risk of blood clots nearly doubles in people with cold agglutinin disease (CAD) compared with that seen in patients with other conditions, a large data study from the U.S. reports. “Increased risk of thrombotic events in cold agglutinin disease: A 10-year retrospective analysis” was published in the…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

The U.S. Food and Drug Administration (FDA) is giving priority review to Sanofi’s request that sutimlimab be approved to treat hemolysis in adults with cold agglutinin disease (CAD). The FDA is expected to announce its decision on or before Nov. 13, as priority review shortens the regulatory process…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

Commercially insured patients with cold agglutinin disease (CAD) in the U.S. use healthcare resources — hospital admissions, outpatient and emergency room visits, and blood transfusions — more frequently than a matched group of patients without the disease, a large real-world study reports. This utilization was also greater before diagnosis,…