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

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Summary

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

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Diseases / indications

• Beta-thalassemia
• Hemoglobinopathies
• Thalassemia

Drugs mentioned

• CTX001

Snippet

Sponsor: Vertex Pharmaceuticals Incorporated | Conditions: Beta-Thalassemia, Thalassemia | Phase 3 | Status: ACTIVE_NOT_RECRUITING