News

The U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz) for the treatment of adults and children ages one month and older with atypical hemolytic uremic syndrome (aHUS), Alexion Pharmaceuticals announced. This is the first time the medication, previously approved to…

Treatment of atypical hemolytic uremic syndrome (aHUS) with Soliris (eculizumab) should be maintained even after clinical deterioration, a case report suggests. This is the first report of suboptimal response to Soliris in a patient with aHUS associated with mutations in both the CFI and THBD genes, the…

Treatment with Soliris (eculizumab) led to successful resolution of atypical hemolytic uremic syndrome (aHUS) that was triggered by inflammatory bowel disease (IBD), a study of two cases has found. The study, “Successful use of eculizumab to treat atypical hemolytic uremic syndrome in patients with inflammatory bowel…

Treatment with Soliris (eculizumab) in pregnant women with atypical hemolytic uremic syndrome (aHUS) may not increase the risk of miscarriage, according to the analysis of a small group of patients enrolled in the Global aHUS Registry. Marie Scully, MD, of University College London, presented the results of the analysis at the…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Two diseases often difficult for doctors to distinguish between — atypical hemolytic uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP) — are both marked by complement system activation and severe deficiency of the ADAMTS13 enzyme, researchers report. These disorders are known to share symptoms, but until now were largely…

Successful kidney transplant for atypical hemolytic uremic syndrome (aHUS) is aided by treatment with Soliris (eculizumab), a small Turkish study has found. The study “Renal Transplantation in Patients With Atypical Hemolytic Uremic Syndrome: A Single Center Experience” was published in the journal Transplantation Proceedings. Hemolytic uremic syndrome (HUS) is…

Activities are set internationally to observe the Sept. 24 aHUS Awareness Day, which aims to raise visibility around the disease, highlight the latest research, and provide insight into patients’ needs. The fifth annual awareness day is coordinated by aHUS Alliance, an international umbrella group of patient organizations…

Soliris (eculizumab) treatment shows a long-term positive benefit-risk profile in both adult and pediatric patients with atypical hemolytic uremic syndrome (aHUS), a registry study reports. The study, “Eculizumab Safety: 5-Year Experience from the Global aHUS Registry,” was published in the journal Kidney International Reports. Soliris, an engineered…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…