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Alexion Pharmaceuticals’ long-acting Ultomiris (ravulizumab) was added to the list of medications available through England’s national health service (NHS) to adults and children weighing at least 10 kilograms (22 pounds), with atypical hemolytic uremic syndrome (aHUS). Ultomiris is Alexion’s successor to Soliris (eculizumab) — also approved for aHUS and…

A child with atypical hemolytic uremic syndrome (aHUS) caused by a mutation in the DGKE gene and with low levels of complement C3 — suggesting complement system dysregulation — responded well to treatment with Soliris…

Ultomiris (ravulizumab) has been approved in Scotland for adults and children weighing at least 10 kg, or 22 lbs, with atypical hemolytic uremic syndrome (aHUS). Ultomiris is Alexion Pharmaceuticals’ successor to Soliris (eculizumab), also approved for aHUS. Both therapies work by blocking the activity of the…

Note: This article was updated to include information from a Hopkins study published on May 5, 2021, of responses after a second vaccine dose was given to transplant patients. Long before COVID-19 changed the world, organ transplant recipients were wearing masks to shield themselves from airborne threats. Immunosuppressive medicines, which often…

Atypical hemolytic uremic syndrome (aHUS) that is triggered by pregnancy is similar to aHUS caused by other triggers, and responds similarly to treatment, a new study suggests. The study, “Pregnancy-triggered atypical hemolytic uremic syndrome (aHUS): a Global aHUS Registry analysis,” was published in the Journal…

Adults with atypical hemolytic uremic syndrome (aHUS) continue to show improvements in kidney function, blood parameters, and quality of life after receiving Ultomiris (ravulizumab) for a median of 1.5 years, new analysis of clinical trial data suggests. The findings were reported in the study “Long-Term Efficacy and…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Soliris (eculizumab) can be used successfully to treat patients with pregnancy-related atypical hemolytic uremic syndrome (aHUS) complicated by kidney, cardiac, and birth complications, as illustrated by a recent case report. The case also highlights the importance of keeping in mind that pregnancy is one of the key triggers of…

Preventive or early treatment with Soliris (eculizumab) appears to increase the chances of a successful kidney transplant among people with atypical hemolytic uremic syndrome (aHUS), a new study reports. While limited by its small sample size, the study found that none of the patients treated with prophylactic (preventive)…