Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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I’m Grateful for Childhood Friends Who Showed They Care
Looking back at my younger self, I don’t think I fully appreciated the help I received from others around me during my school experience. ... Read more -
Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the ... Read more -
Recognizing the Gift of Hemophilia
Hemophilia has been a gift. Let me explain. I know some people will struggle to understand my viewpoint. I realize that they have suffered ... Read more -
Hemophilia Patients Have Better Psychological Well-Being Than Other Adults, Study Reports
People with hemophilia scored better on assessments of psychological well-being than healthy adults, a study found. The finding adds to a growing body of ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
New US Institute for Gene Therapies Seeks to Modernize Regulatory, Reimbursement Framework
The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the ... -
Celebrating Valentine’s Day in Our Own Special Way
With Valentine’s Day over, it’s amazing to look back at the “Love Month” celebrations ... -
BioMarin’s Valrox, Possible 1st Gene Therapy for Hemophilia A, Under FDA Priority Review
The U.S. Food and Drug Administration (FDA) has accepted — under priority review — ... -
Audaire Health and AscellaHealth Partner to Promote Hemophilia Management Program
AscellaHealth and Audaire Health together are offering on a hemophilia management program that aims to containing ...
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Europe’s CHMP Recommends Approval of Hemlibra to Treat Hemophilia A
News
Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds ... Read more