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EP696 : BONE MINERAL DENSITY IS STABLE IN ADULTS WITH PYRUVATE KINASE DEFICIENCY RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT

Researchers

Presenter

  • Hanny Al-Samkari

Principal Investigators

  • Eduard J. van Beers

  • Wilma Barcellini

  • Frdric Galactros

  • Bertil Glader

  • Kevin H. M. Kuo

  • D. Mark Layton

  • Yaddanapudi Ravindranath

  • Hassan Yaish

  • Yan Dong

  • Feng Tai

  • Peter Hawkins

  • Rachael F. Grace

Medical Centers

  • Van Creveldkliniek Department of Internal Medicine,University Medical Center Utrecht,Utrecht,Pays-bas

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

  • University of Utah, Salt Lake City, Utah, United States

  • Unite des Maladies Genetiques du Globule Rouge, CHU Henri Mondor, Creteil, France

  • Wayne State University School of Medicine - Children's Hospital of Michigan, Detroit, MI

  • Division of Cardiology, The Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States,

  • 4Dana-Farber/Boston Children's Cancer and Blood Disorders Center,Boston,United States

  • IRCCS Ca Granda, Ospedale Maggiore Policlinico, Milano, Italy

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

Locations

  • Netherlands

  • United Kingdom

  • United States

  • France

  • Italy

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Musculoskeletal

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

  • Osteoporosis

  • Osteopenia

Biomarkers

  • Bone Mineral Density

Drug/Treatment

  • AG348

Outcome

  • N/A

Study Design

  • Randomized

  • Open Label

Phase

  • II

Study Id's

  • NCT02476916

Sponsors

  • N/A

Result

  • N/A

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