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Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…

To help patients and families in the U.S. facing out-of-pocket medical costs, The Assistance Fund (TAF) has opened a new program for people living with cold agglutinin disease (CAD). This program is designed to provide financial assistance to eligible individuals with medical expenses for FDA-approved treatments, including treatment-related copayments, health…

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

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…