News

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,…

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 autoantibodies found in people with cold agglutinin disease (CAD) carry high levels of sugar molecules, which may help distinguish them from those with related disorders, a study has found. The study, ““Glycosylation of Immunoglobulin Light Chains is Highly Prevalent in Cold Agglutinin Disease,” was published in…

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…

Treatment with rituximab is safe and effective in managing autoimmune hemolytic anemia (AIHA), and seems to be more beneficial for people with warm antibody autoimmune hemolytic anemia (wAIHA) than for those with cold agglutinin disease (CAD), a review study has found. These findings indicate…