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The ataluren (PTC124®) Phase 2a trial in patients with nonsense mutation hemophilia. The trial is a multi-center, open label, dose escalation study. The main goals of the trial are to determine whether treatment with ataluren can result in an increase in Factor VIII or IX levels and whether the drug can safely be given to people with severe hemophilia due to a nonsense mutation. The locations and their status are shown on the map below. As you click on each region, you will be able to see names and locations of specific sites.
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St. Paul's Hospital
Vancouver, BC
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The Bleeding and Clotting Disorders Institute
Peoria, IL
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Indiana Hemophilia and Thrombosis Center
Indianapolis, IN
Principal Investigator:
Amy Shapiro, MD
Contact:
Meadow Heiman
Tel.:
(317) 871-0000
mheiman@IHTC.org
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New England Hemophilia Center, The Medical Center of Central Massachusetts
Worchester, MA
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Children's Hospital of Philadelphia
Philadelphia, PA
Principal Investigator:
Kathy High, M.D. and
Nicole Hasbrouck, M.D.
Contact:
Nicole Hasbrouck, M.D.
Tel.: (215) 590-2232
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio
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Vanderbilt Hemostasis and Thrombosis Clinic
Nashville, TN
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Puget Sound Blood Center
Seattle, WA
Principal Investigator:
Neil Josephson, MD
Contact:
Michelleann A. Bonoan
(206) 398-5942
michelleannb@psbcresearch.org
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