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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)…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

The case of a man with atypical hemolytic uremic syndrome (aHUS) caused by a rare genetic mutation offers insights into the early diagnosis, disease mechanisms, and treatment of this uncommon disorder. Published in the journal Medicine, the case study was titled “Atypical hemolytic uremic syndrome and…

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…