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An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Patients who have symptoms that indicate severe atypical hemolytic uremic syndrome (aHUS) should be diagnosed as quickly as possible so that treatment with Soliris (eculizumab), a potentially life-saving agent, can be initiated promptly, a case study suggests. The study, “Eculizumab for Severe Thrombotic Microangiopathy Secondary to…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

Understanding the disease-related alterations in a large gene cluster that lead to atypical hemolytic uremic syndrome (aHUS) may help in diagnosing and treating the illness, a recent review suggests. The study, “CFHR Gene Variations Provide Insights in the Pathogenesis of the Kidney Diseases Atypical Hemolytic Uremic…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Mutations in the CD46 gene — a regulator of the complement system — may lead to pregnancy-associated atypical hemolytic uremic syndrome (aHUS), according to a case study. These findings may help to identify women at risk of developing the condition. The case report, “Genetic and functional…

Experts in South Korea have proposed a set of new diagnostic criteria to distinguish thrombotic microangiopathy associated with atypical hemolytic uremic syndrome (aHUS). Their study, “Consensus regarding diagnosis and management of atypical hemolytic uremic syndrome,” was published in The Korean Journal of Internal Medicine.

Soliris (eculizumab) maintenance treatment given every three weeks — instead of the standard two weeks — was sufficient to achieve therapeutic blood levels and prevent disease relapse for at least four years in a 2-year-old girl with atypical hemolytic uremic syndrome (aHUS), a case study shows. Soliris’ optimized regimen…

Difficulties in diagnosing atypical hemolytic uremic (aHUS) syndrome in the absence of family history are illustrated in the story of a girl in Sri Lanka who, after multiple diagnoses, was successfully treated for aHUS by long-term plasma exchange, a case study reported. …

Pregnancy-associated atypical hemolytic uremic syndrome (aHUS) usually occurs postpartum, after a pregnancy complication — such as preeclampsia, severe bleeding, and fetal death — and is treated effectively with Soliris (eculizumab), according to a review study. The data highlighted the previously unexplored variety of clinical scenarios in which…