Records containing: Mucopolysaccharidosis Type I (MPS I)
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-03-12A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 Gene Therapy in Participants With Mucopolysaccharidosis Type I
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CLINICAL_TRIALS study record update · 2024-02-14Multi-Centre, Non-Interventional, Double Cohort Study to Assess the Safety of Myozyme® and of Aldurazyme® in Real-World Home Infusion Setting
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CLINICAL_TRIALS study record update · 2019-10-04Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children