Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…
News
A middle-aged woman’s suddenly black fingertips and dark urine were telltale symptoms that she had developed cold agglutinin disease (CAD), according to scientists. Her case was described by a team of Japanese researchers in the study, “Black fingers and dark urine: cold agglutinin disease,”…
A man with cold agglutinin disease (CAD) successfully underwent microvascular free tissue transfer — a tissue and blood vessel transplant — to repair extensive damage to his face, a case study reported. Authors noted that due to the lack of precedence in the literature, this report provides guidance on…
Enjaymo (sutimlimab) quickly led to increases in hemoglobin and lowered the need for blood transfusions in four women with cold agglutinin disease (CAD), in whom the treatment remained safe and effective for about three years, according to data from a Phase 1 open-label extension trial. Trial data was…
Long-term treatment with Sanofi’s Enjaymo (sutimlimab-jome) safely and effectively prevented red blood cell destruction (hemolysis), anemia, and the need for blood transfusions, while improving the quality of life in adults with primary cold agglutinin disease (CAD). That’s according to 2.5 years of data from the Phase 3…
The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…
An elderly woman in Japan is the first reported patient in Asia to develop cold agglutinin disease (CAD) due to COVID-19, according to a new case study. The patient was found to have anemia, or an unusually low red blood cell count, that was not resolved with a blood…
A 71-year-old woman with primary cold agglutinin disease (CAD) was successfully treated with the glucocorticoid prednisone, a case study reported. Its scientists noted that primary CAD typically does not respond to glucocorticoids, but in particular instances like this case, it can. The study, “A Case of Idiopathic Cold…
The case of a man with cold agglutinin disease (CAD) that was found to be secondary to a blood cancer, called diffuse large B-cell lymphoma (DLBCL), was described in a recent report. The researcher proposes that DLBCL should be considered as a potential diagnosis in people with unexplained CAD.
Treatment with Soliris (eculizumab) eased the symptoms of cold agglutinin disease (CAD) in a man in France who possibly developed the condition as a result of a severe infection with SARS-CoV-2, the virus that causes COVID-19, a case study reports. While the patient ultimately died, the researchers said the study’s findings…
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