Use of the investigational therapy sutimlimab results in substantial reductions in patient hospitalizations and blood transfusions among adults with primary cold agglutinin disease (CAD), a post hoc analysis of the CARDINAL trial found. The results of the analysis point…
Cold agglutinin disease (CAD) disease negatively affects a variety of aspects of daily living, with patients often experiencing fatigue, according to new research. In a separate study, it was found that CAD patients appear to be at an increased risk of anxiety or depression. Researchers from Sanofi…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
The U.S. Food and Drug Administration (FDA) has issued a complete response to Sanofi’s application requesting that its investigational therapy sutimlimab be approved for treating hemolysis — the breakdown of red blood cells — in adults with cold agglutinin disease (CAD), the company announced in…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
People with non-Hodgkin’s lymphoma (NHL) — a type of blood cancer — that co-occurs with autoimmune hemolytic anemia (AIHA) have a poor prognosis, suggesting a need for further research into this rare combination of conditions, a case report highlights. The case was reported at the 2020 Lymphoma,…
Apellis Pharmaceuticals and Swedish Orphan Biovitrum (Sobi) are collaborating to advance the development of systemic pegcetacoplan, or APL-2, in the treatment of cold agglutinin disease (CAD) and other disorders, the companies announced in a press release. Pegcetacoplan is an investigational therapy designed to limit activation of…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
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