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The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.

A woman carrying specific gene variants placing her at increased risk of developing atypical hemolytic uremic syndrome (aHUS) was diagnosed with the disease following COVID-19 vaccination, as described in a recent case report. “Given the short time lapse between vaccination … and the clinical manifestations, we believe that vaccine…

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Ultomiris (ravulizumab) was reportedly used for the first time to control atypical hemolytic uremic syndrome (aHUS) in a patient who received a kidney transplant from a living donor, according to a group of doctors in Germany. “We provide first evidence for the efficacy of [Ultomiris] to prevent recurrence…

Soliris (eculizumab) prevented disease recurrence in a small group of patients with primary atypical hemolytic uremic syndrome (aHUS) after they underwent kidney transplantation, a recent study has found. The study, “Efficacy and Safety of Eculizumab in Kidney Transplant Patients With Primary Atypical Hemolytic-Uremic Syndrome,” was published in…

By shortening the time spent on treatment, Ultomiris (ravulizumab), an approved therapy for atypical hemolytic uremic syndrome (aHUS), may be more effective at reducing lost productivity costs compared with its predecessor Soliris (eculizumab), according to a new study. The study, “Quantifying the economic effects of ravulizumab…

Because viral infections can worsen atypical hemolytic uremic syndrome (aHUS), researchers recommend testing patients for infections that are common following an organ transplant. They describe the case of patient, whose aHUS symptoms recurred after undergoing a kidney transplant, in their report, “Concurrent cytomegalovirus enteritis and atypical…

C5a, a terminal product of the complement system, can, by itself, promote blood vessel dysfunction and the subsequent formation of blood clots seen in people with atypical hemolytic uremic syndrome (aHUS), a study shows. Notably, the same C5a-induced molecular mechanisms were detected among patients with severe COVID-19 and whose infection…

For this year’s Rare Disease Day on Feb. 28, the aHUS Alliance is asking patients and caregivers to join them in their project to showcase questions from those affected by atypical hemolytic uremic syndrome (aHUS). This project is aimed at raising awareness for those affected by aHUS.