News

Sanofi’s investigational therapy sutimlimab safely and rapidly prevents red blood cell destruction (hemolysis), increases hemoglobin levels, and reduces fatigue in adults with primary cold agglutinin disease (CAD), according to full data from Part A of the Phase 3 CARDINAL study. These therapeutic benefits were associated with a fast and…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

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 case of a man with COVID-19 who temporarily developed elevated levels of cold agglutinins — the self-reactive antibodies that cause cold agglutinin disease (CAD) — was described in a recent report. According to researchers, this is the third known case of elevated cold agglutinin levels documented in…

Physicians should be aware that patients with autoimmune hemolytic anemia (AIHA) are at greater risk of experiencing thrombotic events — blood vessel obstruction driven by blood clots — to better manage their condition, a case report highlighted. The case study, “Autoimmune Hemolytic…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

An examination of medical records of Indian children with autoimmune hemolytic anemia (AIHA), a group of disorders that includes cold agglutinin disease, found managing infants and those with secondary AIHA challenging, with almost one-third of patients needing treatment…

People with cold agglutinin disease (CAD) have significantly lower blood levels of C3 and C4 — two key molecules of the body’s innate complement immune system — than their healthy peers, a study found. Notably, CAD patients showed the lowest levels of C3 and C4 among autoimmune hemolytic…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…