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Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or ß-thalassaemia: an open-label, multicentre, phase 2 study.
Kuo, Kevin H M; Layton, D Mark; Lal, Ashutosh; Al-Samkari, Hanny; Bhatia, Joy; Kosinski, Penelope A; Tong, Bo; Lynch, Megan; Uhlig, Katrin; Vichinsky, Elliott P.
  • Kuo KHM; Division of Haematology, University of Toronto, Toronto, ON, Canada. Electronic address: kevin.kuo@uhn.ca.
  • Layton DM; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London, UK.
  • Lal A; Division of Hematology, University of California San Francisco Benioff Children's Hospital, Oakland, CA, USA.
  • Al-Samkari H; Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
  • Bhatia J; Agios Pharmaceuticals, Cambridge, MA, USA.
  • Kosinski PA; Agios Pharmaceuticals, Cambridge, MA, USA.
  • Tong B; Agios Pharmaceuticals, Cambridge, MA, USA.
  • Lynch M; Agios Pharmaceuticals, Cambridge, MA, USA.
  • Uhlig K; Agios Pharmaceuticals, Cambridge, MA, USA.
  • Vichinsky EP; Division of Hematology, University of California San Francisco Benioff Children's Hospital, Oakland, CA, USA.
Lancet ; 400(10351): 493-501, 2022 08 13.
Article en En | MEDLINE | ID: mdl-35964609

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Piperazinas / Quinolinas / Talasemia beta / Talasemia alfa Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adult / Female / Humans / Male / Middle aged Idioma: En Año: 2022 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Piperazinas / Quinolinas / Talasemia beta / Talasemia alfa Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adult / Female / Humans / Male / Middle aged Idioma: En Año: 2022 Tipo del documento: Article