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Atypical hemolytic uremic syndrome (aHUS) can co-exist with autoimmune disorders, complicating the diagnosis of the rare blood-clotting disease, a case report shows. The findings highlight the need for genetic testing to diagnose aHUS in complex cases, the researchers said. The case report, “Atypical Hemolytic…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

To help persuade decision-making entities to make the atypical hemolytic uremic syndrome (aHUS) treatment Ultomiris (ravulizumab) available in countries with socially funded healthcare, aHUS Alliance Global Action is asking patients and caregivers worldwide to contribute their experience with the therapy. The organization is seeking input about the…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

Mutations leading to the overactivation of the complement regulator protein C3 should be recognized as a cause of atypical hemolytic uremic syndrome (aHUS), a case report highlights. The report, “Complement C3 mutation causing atypical hemolytic uremic syndrome successfully treated with eculizumab,” was published in the…

Atypical hemolytic uremic syndrome (aHUS) can co-exist with autoimmune thyroid disease, and the two conditions may influence each other’s development, a case report suggests. The report, “Atypical hemolytic uremic syndrome precipitated by thyrotoxicosis: a case report,” was published in BMC Pediatrics. Autoimmune thyroid disease (AITD)…

Self-administering Ultomiris (ravulizumab) as weekly under-the-skin injections may be as safe and effective in preventing overactivation of the complement system as standard intravenous infusions (given directly into the bloodstream), according to interim data from a Phase 3 clinical trial. Excessive activation of the complement system — a set…