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…
Extending the dosing interval of Soliris (eculizumab) maintenance treatment from standard two weeks to three weeks may equally achieve therapeutic blood levels in most adults with atypical hemolytic uremic syndrome (aHUS), a study suggests. The findings are based on a Soliris pharmacokinetic model, which considers its movement…
Ultomiris (ravulizumab) is expected to be more cost-effective than its predecessor Soliris (eculizumab) as a treatment for atypical hemolytic uremic syndrome (aHUS) in the U.S., a new study indicates. The study, “A US cost-minimization model comparing ravulizumab versus eculizumab for the treatment of atypical hemolytic…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
Ultomiris (ravulizumab) is safe and results in stable kidney and blood measurements in children and adolescents with atypical hemolytic uremic syndrome (aHUS) who were previously receiving more frequent infusions of Soliris (eculizumab), a new study indicates. The findings also suggest that pediatric patients could be treated less…
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