Records containing: Lysosomal Storage Disease
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 · 2026-04-07Investigations Into Inborn Errors of Cholesterol Synthesis and Related Disorders
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CLINICAL_TRIALS study record update · 2026-03-16A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)
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CLINICAL_TRIALS study record update · 2025-12-05A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2023-02-13Biomarker for Maroteaux-Lamy Disease: BioMaroteaux-Lamy AN INTERNATIONAL, MULTICENTER, EPIDEMIOLOGICAL PROTOCOL
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CLINICAL_TRIALS study record update · 2017-07-02The Nosology and Etiology of Leukodystrophies of Unknown Cause
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CLINICAL_TRIALS study record update · 2016-10-11Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)
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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 · 2014-08-13A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation
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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 · 2008-03-04Stimulation of the Human Central and Peripheral Nervous System With a Magnetic Stimulator