// App-Quantinova.ai

2583 : Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Researchers

Presenter

  • Rachael F. Grace

Principal Investigators

  • Audra Boscoe

  • Chris Bowden

  • Bertil Glader

  • Hitoshi Kanno

  • D. Mark Layton

  • Eduard van Beers

  • Joan-Lluis Vives Corrons

  • Yan Yan

  • Paola Bianchi

Medical Centers

  • Dana-Farber Cancer Institute/Boston Children's Cancer and Blood Disorder Center, Boston, MA

  • ARIAD Pharmaceuticals, Inc., Cambridge, MA

  • Department of Pediatrics, Stanford School of Medicine, Palo Alto, CA

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

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

  • Department of Hematology, Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands

  • Josep Carreras ICO-Germans and University of Barcelona,Barcelona,Spain

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

Locations

  • United States

  • Japan

  • United Kingdom

  • Netherlands

  • Spain

  • Italy

Companies

  • Agios Pharmaceutical

Study Components

Therapeutic Area

  • Endocrine/metabolic Diseases (ME)

  • Genetic Disorder

Disease

  • Pyruvate kinase (PK) deficiency

Biomarkers

  • Pyruvate

Drug/Treatment

  • N/A

Outcome

  • N/A


Study Design

  • N/A

Phase

  • NA

Study Id's

  • NCT02053480

  • NCT03481738

Sponsors

  • N/A

Result

  • N/A