15,196 results on '"BETA-Thalassemia"'
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2. Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
3. Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
4. A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
5. Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients
6. A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)
7. Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
8. Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
9. A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T)
10. A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (ENERGIZE)
11. Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia
12. A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia
13. 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)
14. CS-101 in Patients With β-thalassemia
15. A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
16. Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
17. Thrombophilia In Beta Thalassemia
18. 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
19. A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major
20. Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
21. A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants
22. Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
23. A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
24. At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
25. ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
26. A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
27. A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
28. Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
29. Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
30. The BENeFiTS Trial in Beta Thalassemia Intermedia (PB04-001)
31. Early Check: Expanded Screening in Newborns
32. Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
33. Long-term Follow-up Study of BRL-101 for TDT
34. Study of the Role of Genetic Modifiers in Hemoglobinopathies (INHERENT)
35. Long-term Follow-up Study of BHC001 for TDT
36. Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children (START)
37. An Open-label Study of a Gene Therapy Product in Beta-Thalassemia
38. Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
39. A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia
40. A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
41. A Long-term Follow-up Study in Participants Who Received CTX001
42. Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
43. Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
44. A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants
45. Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
46. A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel
47. Pilot Study for Patients With Poor Response to Deferasirox
48. Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
49. Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
50. EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
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