Records containing: Lysosomal Storage Diseases
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CLINICAL_TRIALS study results posted · 2026-09-03A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease
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CLINICAL_TRIALS study results posted · 2026-08-06A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing
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CLINICAL_TRIALS study results posted · 2026-08-06A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease
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CLINICAL_TRIALS study record update · 2026-02-09Longitudinal Study of Neurodegenerative Disorders
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CLINICAL_TRIALS study record update · 2026-01-15Identification and Characterization of Novel Coding, Splicing and Non-Coding Variants That Contribute to Genetic Disorders
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CLINICAL_TRIALS study record update · 2026-01-14A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing
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CLINICAL_TRIALS study record update · 2025-09-11A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry
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CLINICAL_TRIALS study record update · 2025-09-05An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).
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CLINICAL_TRIALS study record update · 2024-11-27Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2024-06-12Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score
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CLINICAL_TRIALS study record update · 2023-12-07A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease
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CLINICAL_TRIALS study record update · 2023-11-14Multi-ingredient Supplementation as an Adjunctive Therapy in Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2023-06-05A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease
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CLINICAL_TRIALS study record update · 2023-03-24A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
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CLINICAL_TRIALS study record update · 2022-04-12A Natural History Study of Aspartylglucosaminuria
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CLINICAL_TRIALS study record update · 2021-06-07Complement Activation in the Lysosomal Storage Disorders
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CLINICAL_TRIALS study record update · 2021-05-28Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease: An International, Multicenter, Epidemiological Protocol
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CLINICAL_TRIALS study record update · 2021-05-14A Pilot Study of Diet and Exercise Therapy in Pompe Disease
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CLINICAL_TRIALS study record update · 2021-03-17Natural History Study of Children With Metachromatic Leukodystrophy
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CLINICAL_TRIALS study record update · 2019-01-22Cellular Pharmacodynamics of Small Molecules in Sanfilippo Disease(s) (MPS3) and Other Lysosomal Storage Disorders
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CLINICAL_TRIALS study record update · 2019-01-22Enzymatic and Genotypic Screening of Lysosomal Storage Diseases in Minority Groups
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CLINICAL_TRIALS study record update · 2017-06-29Treatment of Early Infantile-Onset Lysosomal Storage Diseases With Fetal Umbilical Cord Blood (UCB) Transplantation
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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