MUCOPOLYSACCHARIDOSIS II

This is a first-in-human safety study testing a new gene therapy for young boys with a severe form of Hunter syndrome, a rare genetic disorder. Researchers will collect a child's own stem cells, add a working copy of a missing gene in the lab, and return the cells to the child's …

This study continues testing JR-141 to see if it remains safe and effective for Hunter syndrome patients over the long term. It follows 80 patients who were already in the main study, tracking how their thinking skills, walking ability, and organ health change. The goal is to und…

This study continues testing an experimental enzyme replacement therapy called DNL310 in people with Hunter syndrome (MPS II) who completed previous trials. It aims to understand the long-term safety and effectiveness of this treatment when given for up to five years. The therapy…

This study is testing an investigational drug called JR-141 for Hunter syndrome (MPS II), a rare genetic disorder. It aims to see if JR-141 can better control the disease, particularly its effects on brain development and thinking skills, compared to the current standard treatmen…

This study is testing whether a medication called adalimumab can help reduce joint pain and improve movement in children and adults with mucopolysaccharidosis (MPS) types I, II, or VI. These are rare genetic disorders that cause severe joint stiffness and pain. For the first 16 w…

This study aims to gather information by offering expanded newborn screening to 100,000 babies in New York. It will test for 14 rare genetic disorders to see how accurate the tests are, how often these diseases occur, and if finding them early helps children's health. The main go…