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Rachael F. Grace
Audra Boscoe
Chris Bowden
Bertil Glader
Hitoshi Kanno
D. Mark Layton
Eduard van Beers
Joan-Lluis Vives Corrons
Yan Yan
Paola Bianchi
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
United States
Japan
United Kingdom
Netherlands
Spain
Italy
Agios Pharmaceutical
Endocrine/metabolic Diseases (ME)
Genetic Disorder
Pyruvate kinase (PK) deficiency
Pyruvate
N/A
N/A
N/A
NA
NCT02053480
NCT03481738
N/A
N/A