Mucopolysaccharidosis ii

Researchers are testing tividenofusp alfa (DNL310), an experimental enzyme replacement therapy, against the standard treatment idursulfase in children and young adults with mucopolysaccharidosis type II (MPS II). The study includes participants with and without brain involvement,…

This study tests the long-term safety and effects of JR-141, an enzyme replacement therapy, in people with mucopolysaccharidosis II (MPS II), a rare genetic disorder. Participants who completed an earlier JR-141 trial receive the drug again by IV infusion. Researchers track side …

This trial tests whether JR-141, an enzyme replacement therapy given as a weekly IV infusion, can safely manage mucopolysaccharidosis type II (MPS II) over the long term. Researchers will follow about 27 people who completed an earlier JR-141 study, tracking side effects, develop…

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 disorder. About 99 participants who completed earlier studies will receive the drug for up to 5 years. Researchers will monitor side…

This Phase 3 study tests a new drug called JR-141 against the current standard treatment (idursulfase) in 86 people with Hunter syndrome (MPS II). The goal is to see if JR-141 can better reduce harmful substances in the brain and improve thinking skills. Participants can switch t…

This study tests a new 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, modified to produce the missing enzyme, and aims to stop disease progression. Five boys aged 3 to 22 months wi…

This study is an extension of a previous trial testing JR-141, a drug given weekly by IV, for people with Hunter syndrome (a rare genetic disorder). It aims to see if the drug remains safe and effective over a longer period. About 80 participants who completed the earlier study w…

ScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…

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 children and adults aged 5 and older who have significant pain and limited joint motion. Participants receive either adalimumab …

This study follows people who previously received RGX-121, a gene therapy designed to deliver a working copy of the IDS gene to the brain, as a treatment for Mucopolysaccharidosis II (MPS II). The goal is to see how safe the therapy is over the long term and whether it helps with…