Congenital Hemolytic Anemia Treatments

Summary: The study will evaluate the safety and early efficacy of administering the combination of a commercially available potato-based resistant starch along with iron chelation therapy to subjects undergoing alloHCT.

Summary: This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.

Summary: The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity. The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy. It is now common practice to classify patients as having transfusion dependent β-thalassemia (TDT) or non-transfusion-dependent β-thal...