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Soliris (eculizumab) safely and effectively treated children with atypical hemolytic uremic syndrome (aHUS) in a real-world setting, according to a post-marketing surveillance study of pediatric patients in Japan. Notably, nearly three-quarters of the children experienced normalization of all blood parameters in a median of three weeks’ time, the…

A 30-year-old man is thought to have developed atypical hemolytic uremic syndrome (aHUS) after being vaccinated for influenza, as described in a recent case report. “Further studies may be needed to stratify such patients to weigh the risks and benefits of influenza vaccination,” its authors…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Soliris (eculizumab) maintenance therapy administered every three weeks — instead of the biweekly standard regimen — was sufficient to normalize blood parameters and prevent disease recurrence for at least three years in a 4-year-old boy with atypical hemolytic uremic syndrome (aHUS), a case study reported. Individualized Soliris dosing was…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

Genetic testing identified a rare mutation in the complement factor B (CFB) gene that, together with an array of laboratory tests, led to a man being diagnosed with atypical hemolytic uremic syndrome (aHUS). While the CFB mutation left the man genetically predisposed to aHUS, the doctor at Baton Rouge Medical Center in…

Plasma exchange may be doing more harm than good for people with atypical hemolytic uremic syndrome (aHUS), a small study suggests. Specifically, the treatment was found to increase the activation of the complement system — a part of the immune system that is already overly active in aHUS patients.

A 26-year-old man developed atypical hemolytic uremic syndrome (aHUS) triggered by a lung infection and caused by a previously unreported genetic mutation, a case study described. According to researchers, his case highlights the importance of genetic testing for a definitive diagnosis of inherited aHUS. The case study, “A…

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

An 11-year-old boy with atypical hemolytic uremic syndrome (aHUS) became severely ill following infection with SARS-CoV-2, the virus that causes COVID-19, according to a recent case report. “Our study [shows] that severe SARS-COV-2 infection can be developed in pediatric patients with co-existing [aHUS],” the author wrote. The report, “…