News

December 16, 2020December 16, 2020

AstraZeneca Set to Acquire Alexion Pharmaceuticals in 2021

News

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 ... Read more

December 9, 2020December 9, 2020

Specific aHUS Mutations Affect Relapse Risk After Soliris Discontinued, Data Show

News

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 ... Read more

December 2, 2020December 1, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

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 ... Read more

November 26, 2020November 25, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

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 ... Read more

November 25, 2020November 25, 2020

Ultomiris as Shorter Infusion Approved in Europe to Treat aHUS

News

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 ... Read more

November 20, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

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 ... Read more

November 18, 2020November 18, 2020

Most aHUS Patients May Benefit From Dosing Interval Extension in Soliris Maintenance Treatment

News

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 ... Read more

November 11, 2020November 11, 2020

Ultomiris More Cost-effective Than Soliris for aHUS Patients in US, Study Finds

News

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. ... Read more

November 6, 2020November 4, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

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 ... Read more

November 4, 2020November 4, 2020

Ultomiris Effective in Pediatric aHUS Previously Treated With Soliris, Study Suggests

News

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 ... Read more