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…
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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…
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APL-2 Increases Hemoglobin Levels, Reduces Hemolysis in CAD and wAIHA Patients, Trial Results Shows
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,…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
The case report of a patient with cold agglutinin disease (CAD) who developed chronic inflammation of the gums (periodontitis) highlights the need for dentists to be aware of CAD signs and symptoms. The study, “Cold agglutinin disease and its implications for dental treatment,” was published in the journal…
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
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