Tag Archive: Hemophilia A

Sep 20 2018

Pioneering Novel Therapies for Hemophilia

Editor’s Note: Jacqueline Hunter, PhD, a postdoctoral fellow in the Wolfe Laboratory, wrote this article as part of the Advanced Career Exploration (ACE) Fellowship at Children’s Hospital of Philadelphia Research Institute. The ACE program gives fellows the opportunity to pursue projects beyond their main research focus. We’re especially excited to share Dr. Hunter’s work during National Postdoc Appreciation Week.

Normally, the body responds to a bleeding event by forming a clot, which is a complicated process involving multiple proteins in an elegantly orchestrated cascade. When specific proteins of this cascade are absent, one of several debilitating disorders can occur that result in recurrent spontaneous bleeding into the joints and muscles. Researchers at Children’s Hospital of Philadelphia are using clever maneuvering to figure out new therapeutic options for patients with hemophilia A and B.

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Aug 8 2018

New FVIII Center Crosses Disciplines to Reach Answers for Hemophilia A

Hematologists have grappled for many years to understand the basic mechanisms underlying why some young patients with the inherited bleeding disorder hemophilia A develop inhibitory antibodies, also known as factor VIII (FVIII) inhibitors, to protein replacement therapy. The presence of FVIII inhibitors is a serious obstacle that makes it difficult and extremely expensive to treat their hemophilia, leads to complications such as joint disease, and increases mortality.

Such a major challenge requires major brain power. So the National Institutes of Health called on the scientific community to reframe the question: Why is FVIII so immunogenic?

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