Pyruvate kinase deficiency anaemia
Not recruitingStudy on Long-Term Safety of Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children
Study on Long-Term Safety of Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children
- Disease:Pyruvate kinase deficiency anaemia
- Substances:Autologous Cd34+ Hematopoietic Stem Cells Transduced Ex Vivo With A Lentiviral Vector Encoding The Codon-Optimized Version Of Pklr Gene
Spain- 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.














