Records containing: Mucopolysaccharidosis II
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CLINICAL_TRIALS study record update · 2026-06-04A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
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CLINICAL_TRIALS study record update · 2026-04-21An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
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CLINICAL_TRIALS study record update · 2026-04-21A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
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CLINICAL_TRIALS study record update · 2026-04-08Registry of Patients Diagnosed With Lysosomal Storage Diseases
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CLINICAL_TRIALS study record update · 2026-03-27A Phase I-II Study Of Cryopreserved Autologous CD34+ Haematopoietic Stem Cells Transduced Ex Vivo With CD11b Lentiviral Vector Encoding Human IDS Tagged With ApoEII In Patients With Neuronopathic Mucopolysaccharidosis Type II (nMPS II, Hunters Syndrome)
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CLINICAL_TRIALS study record update · 2026-02-11Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics
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CLINICAL_TRIALS study record update · 2026-02-02Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II, and VI
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CLINICAL_TRIALS study record update · 2026-01-21An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects
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CLINICAL_TRIALS study record update · 2025-09-12ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
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CLINICAL_TRIALS study record update · 2025-09-08Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells
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CLINICAL_TRIALS study record update · 2025-08-12Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
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CLINICAL_TRIALS study record update · 2025-08-07A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants With Hunter Syndrome
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CLINICAL_TRIALS study record update · 2025-08-05A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
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CLINICAL_TRIALS study record update · 2025-07-24An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
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CLINICAL_TRIALS study record update · 2025-07-24An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
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CLINICAL_TRIALS study record update · 2025-07-24Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
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CLINICAL_TRIALS study record update · 2024-10-15An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment with Intravenous Drug
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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 · 2024-06-10A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
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CLINICAL_TRIALS study record update · 2024-05-03Long-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the Liver
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CLINICAL_TRIALS study record update · 2023-08-28A Phase 1 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-203 in ERT-Treated Adults With Mucopolysaccharidosis Type II (MPS II) (juMPStart Trial)
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CLINICAL_TRIALS study record update · 2023-02-10Biomarkers for Hunter Syndrome: An International, Multicenter, Observational, Longitudinal Protocol
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CLINICAL_TRIALS study record update · 2022-11-14A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
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CLINICAL_TRIALS study record update · 2022-11-14A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
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CLINICAL_TRIALS study record update · 2022-10-10A Prospective, Observational Study of Pediatric Patients With Neuronopathic Forms of MPS II (Hunter Syndrome)