Peroxisomal single enzyme/protein defect

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 an experimental drug, YOLT-203, in 36 children and adults with primary hyperoxaluria type 1 (PH1), a rare genetic condition that causes painful kidney stones and kidney damage. The goal is to see if a single dose can safely lower oxalate levels in urine and blood…

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 study tests a cream called roflumilast 0.15% for itching in people with mild to moderate atopic dermatitis (eczema). About 40 participants aged 12 and older will apply the cream once daily for 4 weeks. Researchers will track how patients describe their itch and other skin se…

This study investigates how pomegranate dietary supplements influence gut bacteria and inflammation in people with alcohol use disorder and alcohol-related liver disease. Researchers will measure levels of beneficial gut metabolites called urolithins and inflammatory markers in b…

This study aims to validate a blood test for measuring oxalate levels, which is currently not available in Israel. Researchers will compare results from a local lab with an external lab using 50 blood samples. They will also explore how vitamin C levels relate to oxalate in patie…

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 …