Marisa Wexler, MS, senior science writer —

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Atypical hemolytic uremic syndrome (aHUS) that is triggered by pregnancy is similar to aHUS caused by other triggers, and responds similarly to treatment, a new study suggests. The study, “Pregnancy-triggered atypical hemolytic uremic syndrome (aHUS): a Global aHUS Registry analysis,” was published in the Journal…

Preventive or early treatment with Soliris (eculizumab) appears to increase the chances of a successful kidney transplant among people with atypical hemolytic uremic syndrome (aHUS), a new study reports. While limited by its small sample size, the study found that none of the patients treated with prophylactic (preventive)…

Most people with atypical hemolytic uremic syndrome (aHUS) whose disease is in remission after treatment with complement-inhibiting medications like Soliris (eculizumab) can safely stop the therapy with close monitoring, a small study suggests. These findings were reported in “Outcomes of a clinician-directed protocol for discontinuation…

Ultomiris (ravulizumab-cwvz) is safe and effective in children with atypical hemolytic uremic syndrome (aHUS), data from a Phase 3 clinical trial show. These findings were in the study “The long-acting C5 inhibitor, ravulizumab, is effective and safe in pediatric patients with atypical hemolytic uremic syndrome naïve to…

After unanimous votes by the boards of directors of both companies, AstraZeneca has announced it will acquire Alexion Pharmaceuticals. If the acquisition gets all needed regulatory clearances, as well as approvals by the shareholders of both companies, the deal is expected to be finalized in the third quarter…

The specific disease-causing mutations in a person with atypical hemolytic uremic syndrome (aHUS) affect their individual risk of relapse after discontinuing treatment with Soliris (eculizumab), new research shows. aHUS is characterized by abnormal activity of the complement system, a group of immune proteins in the blood. Activation of…

Ultomiris (ravulizumab) is expected to be more cost-effective than its predecessor Soliris (eculizumab) as a treatment for atypical hemolytic uremic syndrome (aHUS) in the U.S., a new study indicates. The study, “A US cost-minimization model comparing ravulizumab versus eculizumab for the treatment of atypical hemolytic…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

Ultomiris (ravulizumab) is safe and results in stable kidney and blood measurements in children and adolescents with atypical hemolytic uremic syndrome (aHUS) who were previously receiving more frequent infusions of Soliris (eculizumab), a new study indicates. The findings also suggest that pediatric patients could be treated less…