The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has …

Sickle Cell Anemia (HbSS)Sickle-β0-thalassemia (HbSβ0)

~Estimated

~$10 – $5,625

Children's Hospital Medical Center, CincinnatiNCT07177300

Phase 1

An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…

Sickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Beta-Thalassemia+1 more

~Estimated

~$10 – $7,500

Daniel BauerNCT06647979

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