Wilson disease

This trial tests an experimental gene therapy called MWAV201 in adults with Wilson disease, a genetic condition that causes copper to build up in the body. The treatment uses a harmless virus to deliver a corrected gene, potentially restoring the body's ability to remove copper. …

This study tests a one-time gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if it is safe and can help the body control copper levels, possibly allowing patients to take fewer or no daily medications. Ab…

This early-stage trial tests a single dose of VTX-801 gene therapy in 4 adults with Wilson's disease, a rare condition where copper builds up in the body. The goal is to see if the treatment is safe and can help control copper levels, possibly allowing patients to stop their usua…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study investigates whether children with Wilson's disease, who appear to have normal heart function, may actually have subtle heart problems. Using advanced echocardiography, ECG, and a blood test for a heart stress marker, researchers will compare these children to healthy …

This study surveys 30 adults with Wilson disease to learn about their daily management, treatment adherence, and interest in gene editing clinical trials. By gathering this information, researchers hope to design better future studies for potential gene therapies targeting specif…

This study will use special PET scans to see how gene therapy (UX704) changes copper distribution in the bodies of 5 people with Wilson disease. The goal is to understand if the therapy helps the liver handle copper better. It is an early, observational imaging study, not a test …