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Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency.
Kuo, Kevin H M; Grace, Rachael F; van Beers, Eduard J; Barcellini, Wilma; Glenthøj, Andreas; Holzhauer, Susanne; Beynon, Vanessa; Morris, Susan; Li, Junlong; Zagadailov, Erin; Patel, Parija; Al-Samkari, Hanny.
Afiliação
  • Kuo KHM; Division of Hematology, University of Toronto, Toronto, Ontario, Canada.
  • Grace RF; Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Harvard Medical School, Boston, Massachusetts, USA.
  • van Beers EJ; Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
  • Barcellini W; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Glenthøj A; Department of Haematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Holzhauer S; Department of Pediatric Hematology and Oncology, Charité University Medicine, Berlin, Germany.
  • Beynon V; Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
  • Morris S; Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
  • Li J; Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
  • Zagadailov E; Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
  • Patel P; Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
  • Al-Samkari H; Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Am J Hematol ; 99(7): 1415-1419, 2024 07.
Article em En | MEDLINE | ID: mdl-38712615
ABSTRACT
Clinically meaningful benefits in the signs, symptoms, and impacts of #PKDeficiency as assessed by disease-specific patient-reported outcome measures were observed in mitapivat-treated adult patients in two phase 3 clinical trials.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Piruvato Quinase / Erros Inatos do Metabolismo dos Piruvatos / Medidas de Resultados Relatados pelo Paciente Idioma: En Ano de publicação: 2024 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Piruvato Quinase / Erros Inatos do Metabolismo dos Piruvatos / Medidas de Resultados Relatados pelo Paciente Idioma: En Ano de publicação: 2024 Tipo de documento: Article