Research.Patient.info
Pyruvate Kinase Deficiency research hub
2 indexed studies · 0 currently recruiting
- active not recruiting
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is gathering information worldwide about a rare blood condition called Pyruvate Kinase Deficiency (PKD). We want to understand how it affects people over time and the treatments they receive, to help improve care in the future.
United States · Canada · Czechia - ongoing, recruitingHuman Pharmacology (Phase I)- Other
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD
This study is testing a new gene therapy for adults with Pyruvate Kinase Deficiency (PKD). It aims to see if the treatment is safe by using the patient's own modified cells to improve red blood cell health.
Spain