Records containing: Hemoglobinopathies
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study results posted · 2026-09-16A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
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CLINICAL_TRIALS study results posted · 2026-07-21Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders