Mucopolysaccharidosis Type Ii
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) [Phase 3]
Phase III study evaluating JR-141 for mucopolysaccharidosis type II is currently active but not recruiting.
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome
Takeda outlines a post-trial access program for idursulfase-IT together with Elaprase to treat children with Hunter syndrome. The update des…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II [Phase 1, Phase 2]
This clinical trial update describes a gene therapy using modified autologous hematopoietic stem cells for Mucopolysaccharidosis type II, cu…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]
Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MP…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: MPS (RaDiCo Cohort) (RaDiCo-MPS)
An update on the RaDiCo-MPS clinical trial cohort sponsored by INSERM, recruiting patients with several mucopolysaccharidosis types I-IX and…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS research · 2026-02-02Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI [Phase 1, Phase 2]
A clinical trial is evaluating the safety and efficacy of adalimumab in mucopolysaccharidosis types I, II, and VI; the study is currently ac…
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CLINICAL_TRIALS research · 2026-01-21Clinical Trial Update: An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects [Phase 3]
An extension phase 3 trial of JR-141 to assess long-term safety and efficacy in Mucopolysaccharidosis type II (Hunter syndrome) patients, wi…
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment [Phase 2, Phase 3]
This clinical trial update describes an extension study of Idursulfase-IT in combination with Elaprase for children and adults with Hunter s…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS research · 2025-08-05Clinical Trial Update: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) [Phase 2, Phase 3]
Clinical trial update for Tividenofusp Alfa (DNL310) vs Idursulfase in pediatric and young adult patients with Mucopolysaccharidosis type II…
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CLINICAL_TRIALS research · 2025-07-24Clinical Trial Update: An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II [Phase 2, Phase 3]
An extension study of JR-141 for Mucopolysaccharidosis Type II is ongoing. The trial covers Phase 2 and Phase 3 and is currently not recruit…
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CLINICAL_TRIALS research · 2025-01-28Clinical Trial Update: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) [Phase 2, Phase 3]
This update covers a Phase 2/3 clinical trial evaluating CAMPSIITE RGX-121 gene therapy for Mucopolysaccharidosis Type II (Hunter syndrome);…
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CLINICAL_TRIALS research · 2014-06-20Clinical Trial Update: The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
This update provides information on the ongoing long-term safety study of Idursulfase-beta in patients with Hunter syndrome (Mucopolysacchar…