News

The Committee for Medicinal Products for Human Use (CHMP) has recommended that a new 100 mg/mL advanced formulation of Ultomiris (ravulizumab) be approved in the European Union to treat atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH). Following this opinion from the CHMP, an arm…

Researchers have identified a novel mutation in the gene DGKE that can cause atypical hemolytic uremic syndrome (aHUS) but is not part of the complement system, suggesting that complement activation does not always underlie the disease. The case report, “Whole exome sequencing revealed a novel…

The aHUS Alliance Global Action estimates there could be 84,000 people living with atypical hemolytic uremic syndrome (aHUS) in the world today — roughly 60,000 more than current estimates — if all patients received the same care as those in the U.S. These numbers were obtained assuming the…

Soliris (eculizumab) is safe and effective for the prevention of recurrence in people with atypical hemolytic uremic syndrome (aHUS) who received kidney transplants, a 10-year follow-up study indicates. The study, “Ten-year outcome of Eculizumab in kidney transplant recipients with atypical hemolytic uremic syndrome–…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Leading up to atypical hemolytic uremic syndrome (aHUS) Awareness Day on Sept. 24, the aHUS Alliance is seeking participation from the global community for a new video project. As in previous years, the nonprofit organization will include submissions in a video and slideshow that will be…

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…