A case of hemolytic anemia and high bilirubin in blood may mean that infection with Epstein-Barr virus (EBV) — the most common one leading to mononucleosis — can cause cold agglutinin disease, even in the absence of typical symptoms of infection, a study suggests. In…
A rare type of cancer called histiocytic sarcoma may trigger the development of cold agglutinin hemolytic anemia, a recent case suggests. This malignant cancer, which arises from the transformation of some immune cells present in the connective tissue, should be considered as a secondary cause of cold agglutinin hemolytic…
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…
Although rare, a new case report highlights that cold type autoimmune hemolytic anemia may be an early manifestation of systemic lupus erythematosus, researchers report. The study, “Cold agglutinin-induced hemolytic anemia as the primary presentation in SLE — A case report,” was published in the Journal of Family…
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
A small molecule targeting a protein of the complement system — a large group of plasma proteins that normally react with one another to fight infection — may help prevent the destruction of red blood cells that characterizes autoimmune hemolytic anemia (AIHA), a study suggests. The study “…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
APL-2, Apellis Pharmaceuticals’ investigational compound for the treatment of autoimmune hemolytic anemia (AIHA), increases the levels of hemoglobin and reduces red blood cells’ destruction (hemolysis) in patients with cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA), a Phase 2 clinical trial shows.
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
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