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Lysosomal Storage Diseases

Clinical trial condition

Lysosomal Storage Diseases

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

710 public trials 130 recruiting studies

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Lysosomal Storage Diseases trial results

Recruiting

Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation

NCT06551961

Conditions: Carbohydrate Metabolism, Inborn Errors, Colonic Diseases, Colonic Diseases, Functional, +15 more
Locations: Nottingham Digestive Disease Centre Nottingham, Nottinghamshire, United Kingdom

Nottingham Digestive Diseases Centre Nottingham, Please Choose..., United Kingdom

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Recruiting

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169

Conditions: Alpha-Thalassemia, Alzheimer Disease, Amyloidosis, +104 more
Locations: xCures Los Altos, California, United States

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Recruiting

Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study.

NCT06607471

Conditions: Anderson-Fabry Disease, Aortic Stenosis, Subvalvular, Aortic Valve Disease, +67 more
Locations: IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

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Recruiting

Development of Health-related Quality of Life Instrument for Patients With Cystinosis

NCT05959668

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations: University Medical Center Hamburg-Eppendorf | Center for Psychosocial Medicine | Department of Medical Psychology Hamburg, Germany

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Recruiting

Gaucherite - A Study to Stratify Gaucher Disease

NCT03240653

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Birmingham Childrens Hospital Birmingham, United Kingdom

New Queen Elizabeth Hospital Birmingham, United Kingdom

Cambridge University Hospital Cambridge, United Kingdom

Great Ormond Street Hospital London, United Kingdom

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Recruiting

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

NCT06081062

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Philippine General Hospital Manila, Philippines

St.Luke's Medical Center Manila, Philippines

Seoul Asan Center Seoul, Songpa-gu, South Korea

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Recruiting

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I

NCT06519552

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: West China Hospital, Sichuan Universit Chengdu, Sichuan, China

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Recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

NCT06207552

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Children's Hospital of Fudan University Shanghai, China

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Recruiting

Natural History of Pompe Disease

NCT03564561

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: Hôpital Raymond Poincaré Garches, Hauts-de-Seine, France

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Recruiting

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

NCT06272149

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine Shanghai, Shanghai Municipality, China

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Lysosomal Storage Diseases

Lysosomal Storage Diseases trial results

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