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Rare disease

Pyruvate kinase deficiency anaemia

  • 4 clinical trials
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4 Clinical trials

Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Mitapivat
  • Sponsor:Agios Pharmaceuticals Inc.
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Rocket Pharmaceuticals Inc.
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Mitapivat
  • Sponsor:Agios Pharmaceuticals Inc.
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Hematopoietic Stem Cells Transduced Ex Vivo With A Lentiviral Vector Encoding The Codon-Optimized Version Of Pklr Gene
  • Sponsor:Rocket Pharmaceuticals Inc.
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