Patricia Inácio, PhD,  science writer—

Intensive treatment with anti-vascular endothelial growth factor (anti-VEGF) medications — commonly used to treat disorders causing vision impairments — are thought to have led to the onset of atypical hemolytic uremic syndrome (aHUS) in a man in his 40s. It is a rare finding in an extremely rare disease,…

Mutations in the THBD gene — previously suggested to predispose people to atypical hemolytic uremic syndrome (aHUS) — might not make any significant contribution to the disease’s development and its severity, according to a small Spanish study. The mutations were found in Spanish aHUS patients at similar frequencies to…

Soliris (eculizumab) was effective in enhancing kidney disease-free survival in patients with atypical hemolytic uremic syndrome (aHUS), a real-world study reported. Specifically, data showed Soliris was able to increase patients’ five-year survival rate without signs of kidney disease by nearly five times. According to authors, this was the…

Pregnancy was identified as the trigger event for atypical hemolytic uremic syndrome (aHUS) that resulted in kidney damage for a genetically predisposed woman in her mid-20s, a U.S. case study reported. The patient had a clinical history of poorly managed high blood pressure and often failed to comply with…

Two new mutations in the FHR2 gene were identified for the first time as the cause of severe atypical hemolytic uremic syndrome (aHUS) in a 24-year-old patient. After two years on dialysis, the man underwent a kidney transplant that was preceded by preventive treatment with Soliris (eculizumab) and…

Maintenance treatment with Soliris (eculizumab) every three to six weeks — instead of the standard two weeks — is sufficient to promote complement inhibition and disease remission in most adults with atypical hemolytic uremic syndrome (aHUS), a small study suggests. This extended-dosing regimen is linked to savings of…

COVID-19 was a strong trigger for relapses of atypical hemolytic uremic syndrome (aHUS) in two brothers who were genetically predisposed to the syndrome, a recent case study reports. In both cases, treatment with Soliris (eculizumab) was effective in alleviating symptoms when given in the early stages of…

A new mutation in the CD46 gene linked to recurrent atypical hemolytic uremic syndrome (aHUS) was identified in a 13-month-old girl, according to a case report from Denmark. As described in the report, the mutation affected the activity of the complement cascade — a part of the immune system…

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…