News

It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

The U.S. Food and Drug Administration (FDA) has approved Enjaymo (sutimlimab-jome) to decrease the need for red blood cell transfusions due to hemolysis, or red blood cell destruction, in adults with cold agglutinin disease (CAD). Enjaymo, which works by preventing hemolysis, is the first and only FDA-approved therapy…

The symptoms of cold agglutinin disease (CAD) often can be overlooked due to their subtlety — particularly in patients with co-existing conditions — a recently reported case of incidental CAD highlights. In the report, researchers in India described a man with a complicated medical history who was admitted on…

Infection by SARS-CoV-2, the virus that causes COVID-19, may trigger cold agglutinin disease (CAD) in adults, according to a small systematic review. mRNa-based vaccines against SARS-CoV-2 were also shown to trigger CAD and other forms of autoimmune hemolytic anemia (AIHA), although only four cases were analyzed. Overall,…

Investment for rare diseases, such as cold agglutinin disease (CAD), has seen a growing interest by investors, outmatching that for biotech and other health segments, according to a recent analysis by Global Genes, a leading rare disease organization. During 2021, a total of $22.9 billion was raised for…

Global Genes is accepting grant applications to fund a series of U.S.-based RARE Meet-Ups for those affected by a rare disease, including cold-agglutinin disease (CAD), scheduled to occur from April to November. Grant applications are being accepted through the Global Genes Grant Portal until Feb. 4. Recipients…

A bone marrow biopsy may be useful as a test for an underlying cancer or other conditions involving the excessive immune cell expansion and guiding treatment in people with newly diagnosed cold agglutinin disease (CAD), according to a new study. The study, “The Role of a…

A case of secondary cold agglutinin disease (CAD) was triggered by a rare, undetected slow-growing type of immune cell cancer in the spleen. The case study, “Cold agglutinin syndrome secondary to splenic marginal zone lymphoma: a case report,” was published in the journal Hematology, Transfusion…

New patient-reported outcome measures capable of adequately capturing and assessing the impact of cold agglutinin disease (CAD) symptoms are needed, a study reported. Its scientists hope that by identifying relevant patient-reported outcome measures, clinicians and others involved in managing patients’ care can gain a better understanding of the concerns…