Dr. Thompson is the chief of the Division of Hematology at CHOP and holds the Elias Schwartz, MD, Endowed Chair in Hematology. Her research focuses on sickle cell disease and thalassemia.
This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused.
A gene in stem cells will then be edited to help the stem cells make more fetal hemoglobin. Higher levels of fetal hemoglobin can improve red blood cell sickling.
The purpose of this research study is to compare transplant (HCT/BMT) to standard of care treatment in young adults with SCD who have experienced complications.
Dr. Chou's research laboratory focuses on regenerative blood cellular therapy, the mechanisms of normal human hematopoietic development, and how these become disrupted in hematologic diseases.
The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease.
