MUCOPOLYSACCHARIDOSIS I
Clinical trials for MUCOPOLYSACCHARIDOSIS I explained in plain language.
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Gene therapy watch: researchers track Long-Term safety of RGX-111 in MPS i patients
Disease control ENROLLING_BY_INVITATIONThis study follows 21 people with MPS I who previously received RGX-111 gene therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior over time to see if the treatment remains safe and continues to help.
Matched conditions: MUCOPOLYSACCHARIDOSIS I
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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Can an arthritis drug ease pain in rare MPS diseases?
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 14 children and adults who have significant pain and limited joint motion. Participants receive either adalimumab or a placebo f…
Matched conditions: MUCOPOLYSACCHARIDOSIS I
Phase: PHASE1, PHASE2 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated May 13, 2026 16:02 UTC