Atypical haemolytic uremic syndrome (aHUS) is an ultra-rare, potentially life-threatening, genetic disease that leads to the formation of blood clots in small blood vessels throughout the body.1,2 These blood clots can lead to stroke, heart attack, kidney failure and death.

aHUS is caused by a permanent genetic mutation that leads to uncontrolled and excessive activation of complement.3 In healthy individuals, complement is used to attack foreign particles; in patients with aHUS, the body is unable to control the activation of complement. As a result, patients with aHUS face a lifelong risk of sudden, potentially catastrophic, and life-threatening complications1,3 that can occur throughout the body and affect vital organs, including the kidneys, heart and brain.4,5

aHUS can be difficult to diagnose because the disease is so rare that many doctors have never encountered a case of it, or may not have recognised it. Initial signs and symptoms of aHUS include confusion, stomach pain, vomiting, and diarrhea. aHUS affects both adults and children. In a large group of aHUS patients, about 60 percent were diagnosed under the age of 18.6 Among pediatric patients, aHUS can cause stroke, heart attacks, seizures and high blood pressure.7 One of the most common signs of aHUS is kidney failure. Historically despite disease management strategies, seventy-nine percent of all patients with aHUS have died, required kidney dialysis, or had permanent kidney damage within three years after diagnosis.3

For more information about aHUS, please visit www.ahussource.eu/patients-caregivers.


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