Records containing: Hurler-Scheie Syndrome
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 · 2026-03-12A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 Gene Therapy in Participants With Mucopolysaccharidosis Type I
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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 · 2023-04-12Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
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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 · 2014-05-05A 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 · 2013-02-21An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2009-08-19A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2009-06-16A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2009-06-16A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
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CLINICAL_TRIALS study record update · 2009-06-16A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old
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CLINICAL_TRIALS study record update · 2009-02-12A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients