Mucopolysaccharidosis I
Clinical trials for Mucopolysaccharidosis I explained in plain language.
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Gene therapy watch: RGX-111 safety tracked in MPS i patients
Disease control By invitation onlyThis study checks the long-term safety of RGX-111, a gene therapy for people with MPS I (a rare genetic disorder). It follows 21 participants who already received the therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior o…
Matched conditions: MUCOPOLYSACCHARIDOSIS I
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:25 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 I
Phase 1/2 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated Jun 27, 2026 09:02 UTC