The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…
News
Researchers found a wide range of demographic, clinical, and laboratory characteristics when studying eight patients with cold agglutinin disease (CAD). The findings revealed that treatment with corticosteroids, surgical removal of the spleen, and the use of CellCept (mycophenolate mofetil) — an antirejection medicine given to transplant patients…
The prevalence of cold agglutinin disease (CAD) is four times higher in colder climates than in warmer areas, according to a study from Europe. The findings also showed that a combination of rituximab with bendamustine is a safe and effective therapy for people with CAD. The study,…
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…
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