Marta Figueiredo, PhD, managing science editor —

Pregnancy is no longer medically unadvised, or contraindicated, as it once was, for women with a history of atypical hemolytic uremic syndrome (aHUS). But it is still considered high risk, an expert emphasized on a recent webinar hosted by ERKNet, the European Rare Kidney Disease Reference Network. The webinar,…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Extending the dosing interval of Soliris (eculizumab) maintenance treatment from standard two weeks to three weeks may equally achieve therapeutic blood levels in most adults with atypical hemolytic uremic syndrome (aHUS), a study suggests. The findings are based on a Soliris pharmacokinetic model, which considers its movement…

Self-administering Ultomiris (ravulizumab) as weekly under-the-skin injections may be as safe and effective in preventing overactivation of the complement system as standard intravenous infusions (given directly into the bloodstream), according to interim data from a Phase 3 clinical trial. Excessive activation of the complement system — a set…

Alexion Pharmaceuticals announced that Ultomiris (ravulizumab) has been approved in the European Union to treat adults and children (weighing at least 10 kgs, or 22 lbs) with atypical hemolytic uremic syndrome (aHUS). “Today’s marketing authorization marks an important step in our efforts to establish Ultomiris as the…

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…

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…

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…

Alexion Pharmaceuticals now holds exclusive rights to Dicerna’s potential RNA interference (RNAi) therapies developed against four targets of the complement system, which plays a role in many diseases, including atypical hemolytic uremic syndrome (aHUS). aHUS results from an abnormal activation of the complement system, a…

Plasma exchange (PEX) therapy given before and after kidney transplants reduces the risk of disease relapse and transplant failure in people with atypical hemolytic uremic syndrome (aHUS), according to an Austrian study. The work also suggested that while treatment with Soliris (eculizumab) may not be necessary for all…