MUCOPOLYSACCHARIDOSIS II

This study tests a one-time gene therapy for boys with Hunter syndrome, a genetic disorder that damages the brain and body. The therapy uses the child's own blood stem cells to produce the missing enzyme, aiming to stop disease progression. Five boys aged 3 to 22 months will be e…

This study looks at the long-term safety and effectiveness of a drug called JR-141 for people with Hunter syndrome, a rare genetic disorder that affects many parts of the body. About 80 participants who completed an earlier study will continue treatment. Researchers will measure …

This study looks at the long-term safety and effects of an experimental drug called DNL310 for people with Hunter syndrome (MPS II), a rare genetic disease. Participants who completed earlier studies can join and receive the drug for up to 5 years. The goal is to see if the drug …

This phase 3 study tests JR-141, an enzyme replacement therapy designed to cross into the brain and treat Hunter syndrome (MPS II). About 86 participants receive the drug to see if it reduces harmful sugar buildup in spinal fluid and improves thinking skills. The goal is to slow …

This study tests whether adalimumab, a drug used for arthritis, can reduce pain and improve joint movement in people with MPS I, II, or VI. It includes 14 children and adults who have significant pain and limited joint motion. Participants receive either adalimumab or a placebo f…

This study offers parents of newborns in select New York hospitals the option to screen their baby for a panel of rare genetic disorders, beyond standard newborn screening. The goal is to learn how accurate the tests are, how common these conditions are, and whether early diagnos…