Thiamine-responsive megaloblastic anemia syndrome

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

This trial will test an artificial intelligence system designed to interpret routine newborn screening tests for inherited metabolic disorders. Researchers will compare the AI's accuracy to standard manual review by trained staff. The study plans to include 200,000 newborns in Ch…

This 10-year observational study will follow 10,000 adults with diabetes, metabolic disorders, and related conditions like high blood pressure and fatty liver disease. Researchers will collect routine medical data to identify markers of disease severity and activity. The goal is …