beti-cel beta thalassemia
Selected indexed studies
- Betibeglogene Autotemcel Gene Therapy for Non-β(0)/β(0) Genotype β-Thalassemia. (N Engl J Med, 2022) [PMID:34891223]
- Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel. (Blood Adv, 2025) [PMID:39418614]
- Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia. (J Mark Access Health Policy, 2021) [PMID:34178295]
_Worker-drafted node — pending editorial review._
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Sources
- Betibeglogene Autotemcel Gene Therapy for Non-β(0)/β(0) Genotype β-Thalassemia. (2022) pubmed
- Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel. (2025) pubmed
- Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia. (2021) pubmed
- Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. (2024) pubmed
- Clinical applications of gene therapy for rare diseases: A review. (2023) pubmed
- In brief: Casgevy for beta thalassemia. (2024) pubmed
- Betibeglogene autotemcel (Zynteglo) for beta thalassemia. (2024) pubmed
- Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia. (2026) pubmed
- β-Thalassemia: evolving treatment options beyond transfusion and iron chelation. (2021) pubmed
- Current therapeutic landscape of β-thalassemia: focus on gene therapy. (2026) pubmed