Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Inclusion criteria

• ✓. At least 6 red blood cell transfusion episodes over a prior 12-month period and Hb levels \<9.5 g/dL in the previous 12 months despite splenectomy OR
• ✓. At least 3 red blood cell transfusion episodes per year over 2 prior years (in the absence of precipitating events such as infection or surgery) and Hb levels \<9.5 g/dL in the previous 12 months despite prior splenectomy.
• ✓. Hb levels \<8.0 g/dL despite prior splenectomy in the absence of transfusions (documented during 2 or more assessments during the prior 1-2 years) regardless of transfusion requirements.