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EP691 : BASELINE CHARACTERISTICS BY AGE OF A GLOBAL COHORT OF PATIENTS DIAGNOSED WITH PYRUVATE KINASE DEFICIENCY – A DESCRIPTIVE ANALYSIS FROM THE PEAK REGISTRY

Researchers

Presenter

  • Paola Bianchi

Principal Investigators

  • Eduard J. van Beers

  • Joan-Lluis Vives Corrons

  • Bertil Glader

  • Andreas Glenthj

  • Hitoshi Kanno

  • Kevin H. M. Kuo

  • D. Mark Layton

  • Vip Viprakasit

  • Yan Yan

  • Rachael F. Grace

Medical Centers

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

  • Metabolic Support UK,Wrexham,United Kingdom

  • Department of Haematology, Hammersmith Hospital, Imperial Healthcare NHS Trust, London, United Kingdom

  • Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

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

  • UOC di Oncoematologia, Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano, Milano, Italy

  • Institute for Leukaemia Research Josep Carreras ENERCA Coordinator,University of Barcelona,Barcelona,Espagne

  • Department of Hematology, Rigshospitalet, Copenhagen, Denmark

  • Department of Transfusion Medicine and Cell Processing, Tokyo Womens Medical University, Tokyo, Japan

  • Department of Pediatrics, Palacky University and University Hospital, Olomouc, Czech Republic

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

Locations

  • Netherlands

  • United Kingdom

  • Thailand

  • United States

  • Italy

  • Spain

  • Denmark

  • Japan

  • Czech Republic

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Prospective

  • Cohort

Phase

  • NA

Study Id's

  • NCT03481738

Sponsors

  • N/A

Result

  • N/A

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