Beta Thalassemia Major
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-02-10Clinical Trial Update: Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major [EARLY_Phase 1]
This is an early-phase (Phase 1) clinical trial evaluating base-edited autologous hematopoietic stem cell transplantation for beta-thalassem…
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CLINICAL_TRIALS research · 2024-12-16Clinical Trial Update: Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia [NA]
A clinical study investigating a 'super transplantation' approach for severe beta-thalassemia using haplo-identical donors is currently recr…
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CLINICAL_TRIALS research · 2024-11-29Clinical Trial Update: Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients [EARLY_Phase 1]
Early-phase clinical trial update evaluating safety and efficacy of β-globin–restored autologous hematopoietic stem cells in β-thalassemia m…
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CLINICAL_TRIALS withdrawal · 2024-10-24Clinical Trial Update: Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies [EARLY_Phase 1]
The Early Phase 1 trial investigating pre-transplant immunosuppression and donor stem cell transplantation for severe hemoglobinopathies has…
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CLINICAL_TRIALS research · 2024-06-20Clinical Trial Update: Inspiratory Muscle Training in Children With Beta Thalasemia [NA]
A clinical trial evaluating inspiratory muscle training in children with beta-thalassemia major is currently recruiting.
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CLINICAL_TRIALS research · 2024-06-14Clinical Trial Update: Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia [NA]
This entry describes a clinical trial evaluating a gene-modified autologous hematopoietic stem cell therapy for transfusion-dependent beta-t…
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CLINICAL_TRIALS trial_result · 2024-03-15Clinical Trial Update: Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children [Phase 4]
A Phase 4 trial evaluating safety and efficacy of early deferiprone treatment in infants and young children with beta-thalassemia major anem…
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FDA approval · 2023-11-14FDA Approved: DEFERASIROX — Supplemental Application (Sun Pharmaceutical Industries, Inc.)
FDA approved a supplemental application for deferasirox submitted by Sun Pharmaceutical Industries, Inc., approving changes to the product l…
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CLINICAL_TRIALS trial_result · 2023-06-18Clinical Trial Update: Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia. [Phase 2]
A Phase 2 trial assessing the safety and tolerability of sotatercept (ACE-011) in adults with beta-thalassemia was terminated; results were …
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FDA approval · 2023-03-16FDA Approved: DEFERASIROX — Supplemental Application (Camber Pharmaceuticals, Inc.)
FDA approved a supplemental application for Deferasirox from Camber Pharmaceuticals, authorizing a generic product under ANDA 216229.
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CLINICAL_TRIALS trial_result · 2023-03-06Clinical Trial Update: PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders [Phase 3]
Phase 3 trial update evaluating the pharmacokinetics of sofosbuvir/ledipasvir in adolescents with hepatitis C infection and beta-thalassemia…
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FDA approval · 2021-11-30FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
FDA approved a supplemental application for Ferriprox (deferiprone) filed by Chiesi USA, Inc.
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CLINICAL_TRIALS trial_result · 2021-08-26Clinical Trial Update: Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation [Phase 2]
Phase 2a pilot study update for NBMI treatment in beta-thalassemia major patients requiring iron chelation.
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FDA approval · 2021-04-30FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
The FDA approved a supplemental application for Ferriprox (deferiprone) from Chiesi USA, Inc., signaling an approved modification to its exi…
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FDA approval · 2021-04-30FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
FDA approved a supplemental application for Ferriprox (deferiprone) from Chiesi USA, Inc., indicating an approved change to the product’s la…
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FDA approval · 2021-03-17FDA Approved: DEFERASOROX — Original Application (Novadoz Pharmaceuticals LLC)
The FDA granted approval for the original ANDA application of Deferasorox (deferasirox) by Novadoz Pharmaceuticals LLC as a generic product.…
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CLINICAL_TRIALS trial_result · 2020-11-05Clinical Trial Update: HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity [Phase 1, Phase 2]
A phase 1/2 clinical trial evaluating reduced intensity conditioning and hematopoietic stem cell transplantation for high-risk hemoglobinopa…
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FDA approval · 2020-06-16FDA Approved: DEFERASIROX — Original Application (Dr. Reddy's Laboratories Inc)
FDA approves Dr. Reddy's Laboratories' deferasirox generic under ANDA210945 for the treatment of chronic iron overload due to blood transfus…
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FDA approval · 2020-06-15FDA Approved: DEFERASIROX — Original Application (Amneal Pharmaceuticals NY LLC)
The FDA approved Amneal's generic deferasirox (Deferasirox) as an original ANDA for the treatment of transfusion-related iron overload in pa…
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FDA approval · 2020-05-19FDA Approved: FERRIPROX — Original Application (Chiesi USA, Inc.)
FDA approved Ferriprox (deferiprone) through the original NDA submitted by Chiesi USA, Inc.
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FDA approval · 2019-12-30FDA Approved: DEFERASIROX — Original Application (Xiromed, LLC)
FDA approved Xiromed, LLC's generic deferasirox (DEFERASIROX) through an original ANDA.
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FDA approval · 2019-05-24FDA Approved: EXJADE — Supplemental Application (Novartis Pharmaceuticals Corporation)
The FDA approved a supplemental application for Exjade (deferasirox) submitted by Novartis Pharmaceuticals Corporation.
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CLINICAL_TRIALS trial_result · 2019-02-25Clinical Trial Update: Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus [Phase 3]
Phase 3 trial update on zinc supplementation in patients with beta-thalassemia major complicated with diabetes mellitus has completed.
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CLINICAL_TRIALS research · 2019-02-20Clinical Trial Update: Sexual Maturation in β-Thalassemia Major Patients in Assiut University Hospital
This clinical trial update examines sexual maturation in beta-thalassemia major patients receiving chelation therapy at Assiut University Ho…
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FDA approval · 2019-02-08FDA Approved: DEFERIPRONE — Original Application (Sun Pharmaceutical Industries, Inc.)
Sun Pharmaceutical Industries received FDA approval for Deferiprone via original ANDA for the iron chelator used to treat iron overload in t…