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Lysosomal Storage Diseases, Nervous System

Clinical trial condition

Lysosomal Storage Diseases, Nervous System

Explore clinical trials studying Lysosomal Storage Diseases, Nervous System. Study availability and eligibility vary by location and protocol.

512 public trials 105 recruiting studies

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Lysosomal Storage Diseases, Nervous System trial results

Not Yet Recruiting

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

NCT07445490

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Département de Neurologie Paris, France

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Not Yet Recruiting

Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease

NCT07351136

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Not specified

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Not Yet Recruiting

Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital

NCT07173010

Conditions: Arthropathy, progressive pseudorheumatoid, of childhood, Bone Diseases, Bone Diseases, Developmental, +36 more
Locations: Not specified

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National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

NCT06985212

Conditions: Acid Sphingomyelinase Deficiency (ASMD), Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations: Groupe Hospitalier Diaconesses Croix Saint-Simon Paris, France

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Not Yet Recruiting

Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease

NCT06869499

Conditions: Blood Platelet Disorders, Brain Diseases, Brain Diseases, Metabolic, +33 more
Locations: Groupe Hospitalier Diaconesses Croix Saint-Simon Paris, France

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Aortic Dimensions in Patients With Fabry Disease

NCT06956573

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Not specified

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The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

NCT06819514

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: General Hospital of Eastern Theater Command Nanjing, Jiangsu, China

Children's Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang, China

First Affiliated Hospital of Zhejiang University Hangzhou, Zhejiang, China

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Treatment Frequency Reduction in Pompe Disease

NCT06575829

Conditions: Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +15 more
Locations: Not specified

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Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

NCT06523517

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Peking union medical college hospital Beijing, China

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Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

NCT06258577

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: China Medical University Hospita Taichung, Taiwan

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Lysosomal Storage Diseases, Nervous System

Lysosomal Storage Diseases, Nervous System trial results

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