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β-thalassemia Major
Clinical trial pipeline · Data from ClinicalTrials.gov
See which β-thalassemia Major trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which β-thalassemia Major trials may be worth asking aboutNorth America
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Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in part…
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evalu…
This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thala…
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material…
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be condu…
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-depende…
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion…