The U.S. Food and Drug Administration (FDA) has approved a new formulation of Ultomiris (ravulizumab), a treatment for adults and children with atypical hemolytic uremic syndrome (aHUS), that allows the medication to be administered in shorter periods of time. The new formulation also was approved for…
Research is needed to identify more mutations that cause atypical hemolytic uremic syndrome (aHUS), as well as to establish best practices for long-term treatment with Soliris (eculizumab), a case report highlights. The study, “A Case Report of Atypical Hemolytic Uremic Syndrome in a Two-Month-Old Infant…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
Ultomiris (ravulizumab) has been approved in Japan for the treatment of adults and children with atypical hemolytic uremic syndrome (aHUS). Developed and marketed by Alexion Pharmaceuticals, Ultomiris is the first and only long-acting inhibitor of the complement C5 protein approved to help patients with…
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…
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