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Hemophilia | Overview

Hemophilia A is a genetic bleeding disorder caused by a mutation in the F8 gene that leads to  low levels of a protein called factor VIII. Factor VIII is necessary for blood clotting. Severe hemophilia A can cause prolonged or spontaneous bleeding, particularly in the muscles and joints, or internal organs. The current standard of care for hemophilia A is to control or prevent bleeding episodes through factor replacement therapy, given by regular intravenous (IV) infusions.

 

Hemophilia A gene therapy clinical trial

Gene Transfer, Dose-Finding Safety, Tolerability, and Efficacy Study of SPK-8011 [a Recombinant Adeno-Associated Viral Vector With Human Factor VIII Gene] in Individuals With Hemophilia A
Recruiting males 18 Years and older (full eligibility criteria)
Boston Children’s Hospital
Started: Dec. 2016
Stacy Croteau, MD, principal investigator
Colleen Dansereau, MSN, RN, CPN617-919-7008 or colleen.dansereau@childrens.harvard.edu

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