Records containing: MPS II
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 · 2026-06-04A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)
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CLINICAL_TRIALS study record update · 2026-03-17PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
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CLINICAL_TRIALS study record update · 2026-02-27A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)
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CLINICAL_TRIALS study record update · 2026-02-09Longitudinal Study of Neurodegenerative Disorders
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CLINICAL_TRIALS study record update · 2026-01-15An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) in Patients With Hunter Syndrome
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CLINICAL_TRIALS study record update · 2024-09-19Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy
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CLINICAL_TRIALS study record update · 2022-09-27A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)
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CLINICAL_TRIALS study record update · 2013-11-07A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy