News

After a year in which COVID-19 was a disruptor, aHUS alliance Global Action is starting 2021 by preparing for Rare Disease Day, research involving family member issues, and a study aimed at learning more about the diagnostic journey of those who have atypical hemolytic uremic syndrome (aHUS). aHUS…

A common mutation in the CD46 gene associated with atypical hemolytic uremic syndrome (aHUS) onset does not seem to be enough to trigger the disorder, as it occurs in both affected and unaffected family members, a genetic analysis shows.  Further analysis found evidence of an…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

The first reported instance of a COVID-19 infection triggering an atypical hemolytic uremic syndrome (aHUS) relapse has been described in a recent case study.  The case highlights the need to monitor aHUS patients, and to continue…

After unanimous votes by the boards of directors of both companies, AstraZeneca has announced it will acquire Alexion Pharmaceuticals. If the acquisition gets all needed regulatory clearances, as well as approvals by the shareholders of both companies, the deal is expected to be finalized in the third quarter…

The specific disease-causing mutations in a person with atypical hemolytic uremic syndrome (aHUS) affect their individual risk of relapse after discontinuing treatment with Soliris (eculizumab), new research shows. aHUS is characterized by abnormal activity of the complement system, a group of immune proteins in the blood. Activation of…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

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…

The European Commission has approved a new intravenous formulation of Ultomiris (ravulizumab) to treat for adults and children with atypical hemolytic uremic syndrome (aHUS) that allows the medication to be given in a shorter period of time. This formulation was also approved for paroxysmal nocturnal hemoglobinuria (PNH),…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…