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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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Comprehensive Bleeding Disorders Center
Peoria, IL
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Indiana Hemophilia and Thrombosis Center
Indianapolis, IN
Principal Investigator:
Anjali Alatkar Sharathkumar, M.D
Contact:
Megan L. Russell
Tel.: (317) 871-0011 Ext.247
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New England Hemophilia Center, The Medical Center of Central Massachusetts
Boston, 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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Puget Sound Blood Center
Seattle, WA
Principal Investigator:
Neil Josephson, M.D.
Contact:
Mark Bray
(206) 292-7069
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Vanderbilt Hemostasis and Thrombosis Clinic
Nashville, TN
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