Marisa Wexler, MS,  senior science writer—

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The case of an infant who developed cold agglutinin disease (CAD) shortly after being vaccinated suggests that intravenous immunoglobulin may be an effective treatment for young children with the autoimmune disorder, according to a new report. The report, “Cold agglutinin disease in an infant: remission after intravenous…

Treatment with the cancer immunotherapy Keytruda (pembrolizumab) can lead to the development of cold agglutinin disease (CAD), the case of a lung cancer patient demonstrates. The report, “Pembrolizumab-Induced Cold Agglutinin Disease,” was published in the American Journal of Case Reports. While the immune system…

Therapies that contain Rituximab may be effective for people with cold agglutinin disease (CAD), a study suggests. The study, “Rituximab-containing therapy for cold agglutinin disease: a retrospective study of 16 patients,” was published in Scientific Reports. CAD is caused by cold agglutinins — a…

A case report from Michigan suggests that coronavirus disease 2019 (COVID-19) could cause cold agglutinin disease (CAD). Results showed that appropriate treatment, including folic acid and a steroid, lowered inflammation and nearly corrected levels of blood cells. The report, “Cold agglutinin disease and autoimmune hemolytic…

The U.S. Food and Drug Administration (FDA) is giving priority review to Sanofi’s request that sutimlimab be approved to treat hemolysis in adults with cold agglutinin disease (CAD). The FDA is expected to announce its decision on or before Nov. 13, as priority review shortens the regulatory process…

The investigational therapy sutimlimab prevented hemolysis, significantly increased hemoglobin, and improved the quality of life in patients with cold agglutinin disease (CAD), according to data from a Phase 3 clinical trial. The results will be presented at the Annual Meeting of the American Society of Hematology in December, in…

Restricted blood flow, or ischemia, in the nose of a cold agglutinin disease (CAD) patient can be successfully treated using a combination that includes aspirin, heparin, nitroglycerin ointment, and hyperbaric oxygen therapy, a case report details. The report, “A case report of severe nasal…

Immune rejection and half-matched donors are risk factors for developing autoimmune hemolytic anemia (AIHA) following blood cancer-treating stem cell transplants, a study found. Titled “Autoimmune hemolytic anemia after allogeneic hematopoietic stem cell transplantation in adults: A southern China multicenter experience,” the study was published in the…