Author Archives: Forest Ray PhD

Sutimlimab continues to lessen the need for blood transfusions, prevent red blood cell destruction, ease fatigue, and limit inflammation in patients with primary cold agglutinin disease (CAD), according to data from two Phase 3 trials. These findings will be presented by Sanofi, the investigational therapy’s developer, at…

Daratumumab eased circulatory symptoms and anemia without causing adverse side effects in a 59-year-old man with cold agglutinin disease (CAD), a recent medical case study reported. The antibody-based medication, normally used to treat myeloma, may offer an alternative to CAD patients who fail to respond to other therapies,…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

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…

Cold agglutinin disease (CAD) seems to be the least common form of autoimmune hemolytic anemia (AIHA) in India, affecting about 15% of patients with the rare disorder, a study has found. The study, “Immunohematological work‐up, severity analysis and transfusion support in autoimmune hemolytic anemia patients,”…

Same but Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

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…

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…

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.