Cirrhosis - dystonia - polycythemia - hypermanganesemia 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 study tests two types of stem cell treatments for people with Parkinsonism, a condition that causes movement and non-movement problems. The goal is to see if these treatments are safe and can improve symptoms like tremors, stiffness, and mood changes. About 120 adults aged 4…

This study tests whether spinal cord stimulation can help people with Parkinson's disease who experience sudden drops in blood pressure when standing up. Forty participants will receive either an implanted stimulator or a non-invasive version. The goal is to see if the treatment …

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 …