MUCOPOLYSACCHARIDOSIS II
Clinical trials for MUCOPOLYSACCHARIDOSIS II explained in plain language.
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New hope for hunter syndrome: Long-Term drug trial launches
Disease control ENROLLING_BY_INVITATIONThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE2, PHASE3 • Sponsor: Denali Therapeutics Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:08 UTC
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New hope for hunter syndrome: Brain-Targeting drug in final testing
Disease control OngoingThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE3 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:03 UTC
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Gene therapy breakthrough offers hope for boys with rare brain disease
Disease control OngoingThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE1, PHASE2 • Sponsor: University of Manchester • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Hunter syndrome drug JR-141 tested for Long-Term safety
Disease control ENROLLING_BY_INVITATIONThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE3 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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New york program offers extra screening for 100,000 newborns
Diagnosis ENROLLING_BY_INVITATIONScreenPlus 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: Albert Einstein College of Medicine • Aim: Diagnosis
Last updated Jun 26, 2026 16:15 UTC
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Can a common arthritis drug ease MPS pain? new trial seeks answers.
Symptom relief OngoingThis 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 …
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE1, PHASE2 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated Jun 27, 2026 09:02 UTC