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S267 : RESULTS FROM A PHASE 2, OPEN-LABEL, MULTICENTER STUDY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

Researchers

Presenter

  • Kevin H.M. Kuo

Principal Investigators

  • D. Mark Layton

  • Ashutosh Lal

  • Hanny Al-Samkari

  • Bo Tong

  • Megan Lynch

  • Katrin Uhlig

  • Elliott P. Vichinsky

Medical Centers

  • St Marys Hospital, Imperial CollegeHealthcare NHS Trust, London, UK

  • Massachusetts General Hospital/Harvard Medical School, Boston, Massachusetts, United States

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • UCSF Benioff Children’s Hospital,Oakland,United States

  • Division of Dermatology, University of Toronto, Toronto,Canada

Locations

  • United Kingdom

  • United States

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Ophthalmology (OP)

Disease

  • Beta-thalassemia

Biomarkers

  • Red Blood Cells

Drug/Treatment

  • Adenosine-5'-triphosphate

  • AG348

Outcome

  • N/A

Study Design

  • Open Label

  • Multicenteric

Phase

  • II

Study Id's

  • NCT03692052

Sponsors

  • N/A

Result

  • N/A

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