Sirnaomics has launched preclinical testing of a first candidate therapy developed with its GalAhead platform for complement-mediated diseases, including atypical hemolytic uremic syndrome (aHUS). The therapy, STP144G, is one of four experimental treatments developed with the proprietary Sirnaomics platform. Data from these early studies are expected to eventually…
COVID-19 might have triggered a relapse of atypical hemolytic uremic syndrome (aHUS) in a 36-year-old woman, according to a recent case report. Despite treatment with Soliris (eculizumab), the patient ultimately died. However, a literature review conducted by study authors revealed nine other cases of COVID-19-associated aHUS and showed…
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…
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