Hemoglobinopathies

• CLINICAL_TRIALS research · 2026-03-13
  Clinical Trial Update: Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders [Phase 2]

  Phase 2 clinical trial evaluating cord blood transplant in children and young adults with blood cancers and select non-malignant disorders; …

• CLINICAL_TRIALS research · 2026-03-03
  Clinical Trial Update: Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) [Phase 3]

  A Phase 3 clinical trial update reports ongoing evaluation of CTX001 for safety and efficacy in pediatric patients with transfusion-dependen…

• CLINICAL_TRIALS research · 2026-01-07
  Clinical Trial Update: Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders [Phase 1, Phase 2]

  A Phase 1/2 clinical trial at Washington University evaluates reduced-intensity conditioning and familial HLA-mismatched bone marrow transpl…

• CLINICAL_TRIALS research · 2025-09-18
  Clinical Trial Update: Prevalence and Hematological Characteristics of Hemoglobinopathies

  This completed clinical trial reports on the prevalence and hematological characteristics of hemoglobinopathies.

• CLINICAL_TRIALS research · 2025-07-24
  Clinical Trial Update: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) [NA]

  ClinicalTrials.gov entry for a second or greater allogeneic hematopoietic stem cell transplant using reduced intensity conditioning, current…

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• CLINICAL_TRIALS research · 2024-07-25
  Clinical Trial Update: Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders [Phase 2]

  Phase 2 clinical trial update describing a reduced-intensity conditioning regimen for patients with non-malignant hematologic disorders, inc…