Records containing: Sly 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 · 2026-04-15Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
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CLINICAL_TRIALS study record update · 2026-04-07ClinicalTrials.gov study NCT03775174
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CLINICAL_TRIALS study record update · 2026-01-07MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
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CLINICAL_TRIALS study record update · 2019-10-16An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old
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CLINICAL_TRIALS study record update · 2019-07-30A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
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CLINICAL_TRIALS study record update · 2018-02-19A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
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CLINICAL_TRIALS study record update · 2017-05-12Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
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CLINICAL_TRIALS study record update · 2011-07-13Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation