Marisa Wexler, MS, senior science writer —

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…

Soliris (eculizumab) can meaningfully ease fatigue and other symptoms commonly reported by people with atypical hemolytic uremic syndrome (aHUS), a study drawn from real-world data shows. These benefits also appear to be sustained with continued treatment, findings suggested. The study, “Functional Assessment of Fatigue and Other…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

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…

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…