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After the successful treatment with Soliris (eculizumab) of a 20-year-old man experiencing a flare-up of atypical hemolytic uremic syndrome (aHUS), his medical team called for the therapy to be made available to all aHUS patients, despite its high cost. “[Soliris] should be started early if plasmapheresis [plasma exchange…

Soliris (eculizumab) safely and effectively treated chronic kidney disease in children with atypical hemolytic uremic syndrome (aHUS), a review study confirmed. Researchers suggested that more research is necessary, however, to establish its effectiveness, safety, and time of discontinuation in the long term. The review study, “The treatment…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

Soliris (eculizumab) safely and effectively treated a severe episode of atypical hemolytic uremic syndrome (aHUS) in an adolescent with another autoimmune disease called systemic lupus erythematosus (SLE), a case report described. The therapy’s use also was safely discontinued, without an aHUS relapse. The case study, “Successful Discontinuation…

Children who develop atypical hemolytic uremic syndrome (aHUS) due to autoantibodies may have an upper airway or digestive tract infection as an initial symptom, according to a report detailing eight such pediatric aHUS cases in China. Likely treatment could involve the use of plasma exchange, corticosteroids, and immunosuppressive agents, its…

Pregnancy triggered atypical hemolytic uremic syndrome (aHUS) in a genetically predisposed 18-year-old woman, causing rapid kidney failure two years after her child’s birth, researchers in the U.S. have reported. The woman’s kidney failure was initially thought to be due to high blood pressure. However, after genetic testing, she was…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

As a young boy in Ireland, Ronald Dixon was drawn to how sunlight made stained glass art come alive. The son of an organist and choirmaster, Dixon spent much of his childhood in church, for years admiring the craftsmanship of those who created the work that captured his imagination. Dixon…

Uterine fibroid surgery triggered atypical hemolytic uremic syndrome (aHUS) in a woman without disease-related genetic defects, a case study reported. Clinicians should investigate conditions such as aHUS after major gynecologic surgery accompanied by kidney impairment and abnormal lab tests indicating red blood cell damage to prevent irreversible kidney disease,…

Eight cases of atypical hemolytic uremic syndrome (aHUS) that occurred in people with underlying conditions that could have prompted increased activity of the complement cascade — a part of the immune system that is thought to drive aHUS — were described in a recent report. “The cases described…