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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…

Soliris (eculizumab) withdrawal after several months of treatment and full recovery in people with atypical hemolytic uremic syndrome (aHUS) is both safe and cost effective, a study suggests. The study, “Atypical haemolytic uraemic syndrome in the eculizumab era: presentation, response to treatment and evaluation of an eculizumab…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…

A multidisciplinary approach is needed to evaluate patients suspected of having atypical hemolytic uremic syndrome (aHUS) and to select the best course of treatment in each instance, a case report says. The report, “A Multimodality Approach to Assessing Factor I Genetic Variants in Atypical Hemolytic Uremic Syndrome,” was…

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…