Records containing: Mucopolysaccharidosis 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 · 2026-04-08Registry of Patients Diagnosed With Lysosomal Storage Diseases
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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 · 2025-09-17A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China
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CLINICAL_TRIALS study record update · 2025-08-28An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I
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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 · 2025-03-19Phase I/II Study of JR-171 ㏌ Patients With Mucopolysaccharidosis Type I
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CLINICAL_TRIALS study record update · 2024-10-30Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products at St. Jude Children's Research Hospital
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CLINICAL_TRIALS study record update · 2024-07-03A Long-term Follow-up Study to Evaluate the Safety of RGX-111
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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 · 2024-02-20A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme® (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
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CLINICAL_TRIALS study record update · 2023-10-31An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2023-03-15An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181
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CLINICAL_TRIALS study record update · 2023-03-15A Two-Stage, Phase 1/2, Open-Label Study of the Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)
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CLINICAL_TRIALS study record update · 2023-02-21A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)
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CLINICAL_TRIALS study record update · 2022-10-25A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders
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CLINICAL_TRIALS study record update · 2019-07-11Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)
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CLINICAL_TRIALS study record update · 2018-09-25An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102
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CLINICAL_TRIALS study record update · 2016-01-26A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2014-11-24Phase II/III, Randomized, Clinical Trial of the Effects of Nutropin AQ® on Growth and Bone Metabolism in Children With MPS I, II, and VI and Short Stature
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CLINICAL_TRIALS study record update · 2014-07-02A Trial of Antigen-specific Immune Tolerance Induction in Mucopolysaccharidosis I (MPS I) Patients Initiating Enzyme Replacement Therapy With Aldurazyme® (Laronidase)
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CLINICAL_TRIALS study record update · 2014-05-05A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).
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CLINICAL_TRIALS study record update · 2013-05-24Unrecognized Mucopolysaccharidosis I, II, IVA, and VI in the Pediatric Rheumatology Population
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CLINICAL_TRIALS study record update · 2013-02-21An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2013-02-21A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I