Beta-thalassemia
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-06-12EMA Authorised: Deferasirox Mylan (deferasirox) — Mylan Pharmaceuticals Limited
The EMA has authorised Deferasirox Mylan for iron overload indications, including beta-thalassemia, with Mylan Pharmaceuticals Limited as th…
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EMA approval · 2026-04-21EMA Authorised: Ferriprox (Deferiprone) — Chiesi Farmaceutici S.p.A.
The European Medicines Agency authorised Ferriprox (deferiprone) from Chiesi Farmaceutici S.p.A. for the treatment of beta-thalassemia and i…
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
NIAID-sponsored clinical trial update indicates recruitment is ongoing to collect human biospecimens for basic and clinical research into gl…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia [Phase 1, Phase 2]
A Phase 1/2 clinical trial of ALS20-101, a lentiviral gene therapy for beta-thalassemia, is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) [Phase 3]
A Phase 3 pediatric study evaluating mitapivat for transfusion-dependent alpha- and beta-thalassemia to assess efficacy, pharmacokinetics, a…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia [Phase 3]
This clinical trial update describes a Phase 3 study to evaluate the efficacy, pharmacokinetics, and safety of mitapivat in pediatric partic…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) [Phase 2]
This is a Phase 2 clinical trial evaluating an immune suppression treatment for people with sickle cell disease or beta-thalassemia undergoi…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients
This research study from Assiut University investigates how microRNA-155 expression relates to the formation of alloantibodies in transfusio…
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Long-term Follow-up Study in Participants Who Received CTX001 [Phase 3]
A long-term follow-up update on participants who received CTX001 in a Phase 3 study, reporting safety and durability data for beta-thalassem…
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CLINICAL_TRIALS research · 2026-03-23Clinical Trial Update: Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease [Phase 3]
Phase 3 clinical trial update for CTX001 (exagamglogene autotemcel) assessing a single dose in transfusion-dependent beta-thalassemia and se…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia [Phase 1, Phase 2]
NHLBI-sponsored clinical trial update describes adding JSP191, a C-kit antibody, to a nonmyeloablative hematopoietic cell transplantation re…
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CLINICAL_TRIALS research · 2026-03-03Clinical 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…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia [Phase 2]
This document announces a Phase 2 clinical trial evaluating the safety and efficacy of AND017 in patients with beta-thalassemia, currently r…
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CLINICAL_TRIALS trial_result · 2026-02-23Clinical Trial Update: A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
A real-world clinical trial is ongoing to evaluate luspatercept in adults with transfusion-dependent beta-thalassemia in the Middle East; th…
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EMA approval · 2026-02-19EMA Authorised: Casgevy (Exagamglogene autotemcel) — Vertex Pharmaceuticals (Ireland) Limited
EMA authorised Casgevy (exagamglogene autotemcel) by Vertex for beta-thalassemia and sickle cell disease, with conditional approval and orph…
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CLINICAL_TRIALS trial_result · 2026-02-12Clinical Trial Update: A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia [EARLY_Phase 1]
An early-phase trial evaluating CS-101 in Beta-thalassemia has completed. The study assessed the safety and preliminary efficacy of CS-101 i…
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CLINICAL_TRIALS research · 2026-02-10Clinical Trial Update: A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
A follow-up clinical trial update evaluating the long-term safety and efficacy of CS-101 injections in participants with Beta-thalassemia, w…
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CLINICAL_TRIALS research · 2026-02-10Clinical Trial Update: A Long-term Follow-up Study in Participants Who Received CS-101
This clinical trial update reports a long-term follow-up study of participants who received CS-101 for beta-thalassemia. Enrollment is by in…
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CLINICAL_TRIALS other · 2026-02-10Clinical Trial Update: CS-101 in Patients With β-thalassemia [EARLY_Phase 1]
An early-phase 1 clinical trial update for CS-101 in patients with Beta-thalassemia; recruitment is ongoing by invitation.
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EMA approval · 2026-02-05EMA Authorised: Exjade (deferasirox) — Novartis Europharm Limited
The EMA authorised Exjade (deferasirox) by Novartis Europharm Limited for beta-thalassemia and iron overload in the European Union.
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CLINICAL_TRIALS research · 2026-02-03Clinical Trial Update: Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies [Phase 1]
Phase 1 clinical trial evaluating hematopoietic stem cell BCL11A enhancer gene editing for severe sickle cell disease and beta-thalassemia, …
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CLINICAL_TRIALS research · 2026-01-13Clinical Trial Update: Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients [Phase 2]
A Phase 2 clinical trial evaluating the efficacy and safety of luspatercept in combination with thalidomide for transfusion-dependent beta-t…
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CLINICAL_TRIALS research · 2026-01-13Clinical Trial Update: Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia [Phase 1, Phase 2]
BRL-101 is in Phase 1/2 trials for transfusion-dependent beta-thalassemia and is currently enrolling by invitation.
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CLINICAL_TRIALS research · 2026-01-06Clinical Trial Update: Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia [Phase 2]
This is a Phase 2 pediatric safety and pharmacokinetics study of luspatercept (ACE-536) in children with beta-thalassemia, currently recruit…
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia [Phase 2]
A Phase 2 clinical trial is investigating the combination of thalidomide and hydroxyurea in patients with transfusion-dependent beta-thalass…