News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Treatment with Soliris (eculizumab) successfully prevented the recurrence of atypical hemolytic uremic syndrome (aHUS) in a 64-year-old woman who had received a kidney transplant, without compromising her recovery from COVID-19, a case study reports. The study, “COVID-19 in a kidney transplant recipient treated with eculizumab for…

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…