MUCOPOLYSACCHARIDOSIS II
Clinical trials for MUCOPOLYSACCHARIDOSIS II explained in plain language.
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Hope for hunter syndrome: Long-Term brain treatment study continues
Disease control ENROLLING_BY_INVITATIONThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE3 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC
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Five-Year hunt for hunter syndrome treatment safety
Disease control ENROLLING_BY_INVITATIONThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE2, PHASE3 • Sponsor: Denali Therapeutics Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:07 UTC
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New Brain-Targeting drug enters final testing for rare genetic disease
Disease control OngoingThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE3 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Mar 20, 2026 14:47 UTC
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New hope for easing debilitating pain in rare genetic diseases
Symptom relief OngoingThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE1, PHASE2 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated Mar 23, 2026 15:16 UTC
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Massive study screens 100,000 babies for hidden rare diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis 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…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC