A diagnostic process combining genetic, clinical, and patient inquiry data may help in the identification and categorization of patients with suspected atypical hemolytic uremic syndrome (aHUS), a study suggests. The new scheme already has aided in identifying a new, likely disease-contributing gene variant for aHUS.
Soliris (eculizumab) can effectively treat patients with both atypical hemolytic uremic syndrome (aHUS) and C3 glomerulopathy (C3G), two disorders that are known to impair kidney function, according to a case report. The study, “…
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…
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
Ultomiris (ravulizumab) can safely and effectively treat women with atypical hemolytic uremic syndrome (aHUS) whose symptoms are triggered by pregnancy, a Phase 3 clinical trial analysis reveals. The study, “Efficacy and safety of the long-acting…
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…
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…
COVID-19 was identified as the trigger for atypical hemolytic uremic syndrome (aHUS) in two adults, but the patients — one man and one woman — responded well to treatment with Soliris (eculizumab), according to a recent study. The study, “Case series: coronavirus disease 2019 infection as a…
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
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