News

A case of cold agglutinin disease (CAD) secondary to bacterial infection in a young woman in the U.K. was initially mistaken for blood cancer, according to a recent report. The researchers noted that the 27-year-old patient had severe hemolytic anemia, a condition in which red blood cells are…

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

COVID-19 can worsen symptoms of cold agglutinin disease (CAD), a recent case report highlights. The scientists stressed the need for further research to determine the best ways to care for people with new or worsening CAD that develops in the context of a COVID-19 infection. In this case, treatment…

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

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.