Hurler-scheie Syndrome
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: RGX-111 Gene Therapy in Patients With MPS I [Phase 1, Phase 2]
The RGX-111 gene therapy trial for Mucopolysaccharidosis Type I is currently suspended in Phase 1/2.
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CLINICAL_TRIALS trial_result · 2014-05-05Clinical Trial Update: A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients [Phase 4]
Phase 4 study investigating the relationship between anti-laronidase antibodies and urinary glycosaminoglycans in Aldurazyme-treated patient…
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FDA approval · 2013-04-03FDA Approved: ALDURAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Aldurazyme (laronidase) from Genzyme Corporation. This constitutes a supplemental approval of th…
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CLINICAL_TRIALS trial_result · 2013-02-21Clinical Trial Update: Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I [Phase 1]
Extension study of intrathecal enzyme replacement therapy for Mucopolysaccharidosis Type I (Phase 1) has been terminated. The trial involved…
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CLINICAL_TRIALS trial_result · 2009-08-19Clinical Trial Update: Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I [Phase 3]
Phase 3 study of Aldurazyme in patients with mucopolysaccharidosis type I (Hurler and Hurler-Scheie) has completed, with results posted in 2…
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CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old [Phase 2]
Phase 2 study evaluating the safety and pharmacokinetics of Aldurazyme (laronidase) in patients under 5 years old with mucopolysaccharidosis…
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CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients [Phase 3]
This Phase 3 extension study evaluated the safety and efficacy of Aldurazyme (laronidase) in patients with Mucopolysaccharidosis I and has c…
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CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease [Phase 4]
Phase 4 dose-optimization study of Aldurazyme (Laronidase) in patients with Mucopolysaccharidosis I completed with results posted in 2009.
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CLINICAL_TRIALS trial_result · 2009-02-12Clinical Trial Update: Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease [Phase 3]
Phase 3 study of Aldurazyme (laronidase) for mucopolysaccharidosis I has completed, with results posted in 2009.