MUCOPOLYSACCHARIDOSIS II

This study tests a new gene therapy for young boys with Hunter syndrome, a genetic disorder that damages the brain and body. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to stop disease progression. Five boys aged 3 to 22 month…

This phase 3 study tests a drug called JR-141 in 86 people with Hunter syndrome (MPS II), a rare genetic disease that damages the body and brain. The goal is to see if the drug can reduce harmful substances in the spinal fluid and improve thinking and learning skills. Participant…

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. About 80 participants who completed an earlier study will continue treatment. Researchers will track changes in brain function, thinking sk…

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. About 99 participants who finished earlier studies will receive the drug for up to 5 years. The goal is to see if the drug …

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 gives parents the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. Researchers will test 100,000 babies born in New York hospitals to see how accurate the screening is and how often these conditions…