News

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

A 6-month-old girl who was diagnosed with cystic fibrosis (CF) went into kidney failure due to atypical hemolytic uremic syndrome (aHUS), according to scientists who worked on her case report. Her kidney function was completely restored following hemodialysis, plasma exchange therapy — a form of treatment that replaces…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

Soliris (eculizumab) is effective for treating relapses in patients with atypical hemolytic uremic syndrome, even in the presence of severe kidney disease, according to a case report. The case study describes a 29-year-old woman with aHUS who experienced a relapse complicated by severe kidney disease, after recovering from…

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…