Beta-thalassemia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2025-12-17Clinical Trial Update: A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia [Phase 2, Phase 3]
A Phase 2/3 safety and efficacy study of CTX001 in transfusion-dependent beta-thalassemia has completed.
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CLINICAL_TRIALS research · 2025-11-25Clinical Trial Update: An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia [Phase 1, Phase 2]
An open-label Phase 1/2 trial evaluating Vebeglogene Autotemcel for transfusion-dependent beta-thalassemia. The study is currently recruitin…
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EMA approval · 2025-11-24EMA Authorised: Reblozyl (Luspatercept) — Bristol Myers Squibb Pharma EEIG
The European Medicines Agency authorised Reblozyl (luspatercept) by Bristol Myers Squibb Pharma EEIG for treating anemia associated with bet…
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CLINICAL_TRIALS trial_result · 2025-11-12Clinical Trial Update: Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Sangamo Therapeutics provides an update on long-term follow-up for sickle cell disease and beta-thalassemia subjects previously treated with…
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CLINICAL_TRIALS research · 2025-11-12Clinical Trial Update: A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials [Phase 3]
This clinical trial update describes a Phase 3 study evaluating the long-term safety of luspatercept in participants who have previously par…
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FDA approval · 2025-10-21FDA Approved: DEFERASIROX — Supplemental Application (Actavis Pharma, Inc.)
FDA approved a supplemental application for Deferasirox from Actavis Pharma, Inc. This action updates the regulatory status of the product.
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CLINICAL_TRIALS research · 2025-10-20Clinical Trial Update: A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India [Phase 4]
Phase 4 clinical trial evaluating the safety and effectiveness of luspatercept for transfusion-dependent anemia associated with myelodysplas…
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CLINICAL_TRIALS research · 2025-10-06Clinical Trial Update: Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With β-TTD [NA]
A clinical trial is currently recruiting to evaluate physiological and psychological factors involved in exercise intolerance in patients wi…
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EMA approval · 2025-09-30EMA Authorised: Deferasirox Accord (deferasirox) — Accord Healthcare S.L.U.
The EMA authorised Deferasirox Accord (deferasirox) for treating iron overload and beta-thalassemia.
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EMA approval · 2025-09-29EMA Authorised: Deferasirox Mylan (deferasirox) — Mylan Pharmaceuticals Limited
EMA has authorised Deferasirox Mylan (deferasirox) for treatment of iron overload, including in patients with beta-thalassemia. This marks E…
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CLINICAL_TRIALS research · 2025-09-24Clinical Trial Update: A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes [Phase 2]
A Phase 2 clinical trial update for SP-420 in transfusion-dependent beta-thalassemia and low-risk myelodysplastic syndromes, currently recru…
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CLINICAL_TRIALS research · 2025-09-24Clinical Trial Update: YOLT-204 in Patients With Hemoglobinopathies [EARLY_Phase 1]
This is an early Phase 1 clinical trial update for YOLT-204 in patients with hemoglobinopathies, including transfusion-dependent beta-thalas…
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: A Long-term Follow-up Study in Participants Who Received CS-101 [NA]
A long-term follow-up study of participants who received CS-101 for beta-thalassemia is ongoing, with the study currently recruiting.
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CLINICAL_TRIALS research · 2025-09-05Clinical Trial Update: Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia [Phase 3]
Phase 3 clinical trial evaluating the combination of N-Acetyl Cysteine and Alpha Lipoic Acid in Beta-thalassemia is currently not recruiting…
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CLINICAL_TRIALS research · 2025-08-12Clinical Trial Update: A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. [Phase 2]
This is a Phase 2 clinical trial evaluating luspatercept (ACE-536) in Chinese participants with beta-thalassemia who require regular red blo…
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CLINICAL_TRIALS research · 2025-08-08Clinical Trial Update: AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion [Phase 2]
A Phase 2 clinical trial evaluating allogeneic stem cell transplantation with alpha/beta T cell and CD19+ B cell depletion for malignant and…
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CLINICAL_TRIALS trial_result · 2025-08-05Clinical Trial Update: Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. [NA]
This update describes ongoing clinical evaluation of KL003 Cell Injection for safety and efficacy in patients with transfusion-dependent bet…
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CLINICAL_TRIALS research · 2025-08-05Clinical Trial Update: Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients [NA]
The document discusses a lentiviral vector gene therapy being evaluated for beta-thalassemia. The trial is currently recruiting.
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CLINICAL_TRIALS trial_result · 2025-08-01Clinical Trial Update: Impact of Virtual Reality on Pain and Anxiety During Intravenous Cannulation Procedures Among Thalassemia Patients in UAE. [NA]
A completed clinical trial evaluated whether virtual reality reduces pain and anxiety during intravenous cannulation in beta-thalassemia pat…
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CLINICAL_TRIALS trial_result · 2025-05-30Clinical Trial Update: Long Term Beta Thalassemia Treatment: Findings From The Extension Period [Phase 2]
Long-term extension-period findings from a Phase 2 trial evaluating treatment for beta-thalassemia are reported.
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CLINICAL_TRIALS trial_result · 2025-05-25Clinical Trial Update: A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) [Phase 3]
Phase 3 trial evaluating the efficacy and safety of mitapivat in transfusion-dependent alpha- and beta-thalassemia; results have been posted…
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CLINICAL_TRIALS trial_result · 2025-04-02Clinical Trial Update: EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) [Phase 1, Phase 2]
Editas Medicine reports an update for EDIT-301, an autologous hematopoietic stem cell gene-editing therapy, currently in Phase 1/Phase 2 for…
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CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia [Phase 1]
A Phase 1 safety and efficacy study evaluating ET-01 for transfusion-dependent beta-thalassemia was terminated.
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CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia. [NA]
A study evaluating ET-01 transplantation for transfusion-dependent beta-thalassemia has been terminated.
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CLINICAL_TRIALS research · 2025-03-10Clinical Trial Update: Β-Thalassemia Treatment with KL003 Cell Injection [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating KL003 cell injection for transfusion-dependent beta-thalassemia is currently recruiting.