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P1544 : BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT

Researchers

Presenter

  • Hanny Al-Samkari

Principal Investigators

  • Rachael F. Grace

  • Andreas Glenthj

  • Oliver Andres

  • Wilma Barcellini

  • Frdric Galactros

  • Kevin H. M. Kuo

  • D. Mark Layton

  • Marta Morado Arias

  • Vip Viprakasit

  • Yan Dong

  • Feng Tai

  • Liz Grekas

  • Karishma Khoja

  • Sarah Gheuens

  • John B. Porter

  • Eduard J. van Beers

Medical Centers

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Haematology Department, University College London Hospitals, London, UK

  • Department of Paediatrics,University of Wurzburg,Wurzburg,Germany

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

  • Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University,Bangkok,Thaïlande

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • Hem

  • Haematology Department, University College London Hospitals, London, UK

  • Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University,Bangkok,Thaïlande

  • Dana-Farber/Boston Children's Cancer...

  • Department of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Haematology Department,University College London Hospitals,London,Vereinigtes Königreich

  • Dana-Farber/Boston Childrens Hospital Cancer and Blood Disorders Center, Boston, MA

  • Division of Hematol

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

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

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Division of Gynecologic Oncology, Massachusetts General Hospital, Harvard Medical School, Boston, MA

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

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

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Vereinigtes Königreich

  • Department of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Royaume-uni

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

  • Agi

  • Haematology Department, University College London Hospitals, London, UK

  • Department of Medical Oncology, University College London Hospital, London, United Kingdom

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

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

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

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

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

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

  • Department of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark

  • Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University,Bangkok,Thaïlande

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Royaume-uni

  • Department of Paediatrics,University of Wurzburg,Wurzburg,Germany

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

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • Department of Hematology,

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Vereinigtes Königreich

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Division of General Surgery, University of Toronto, Toronto, ON, Canada

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University,Bangkok,Thaïlande

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

Locations

  • United States

  • Denmark

  • Germany

  • Italy

  • France

  • Canada

  • United Kingdom

  • Uruguay

  • Spain

  • Thailand

  • Netherlands

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Musculoskeletal

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

  • Osteoporosis

  • Osteopenia

Biomarkers

  • Bone Mineral Density

  • Red Blood Cells

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Placebo Control

  • Cross Over

Phase

  • N/A

Study Id's

  • NCT02476916

  • NCT03548220

  • NCT03559699

  • NCT03853798

Sponsors

  • N/A

Result

  • N/A

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