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Lysosomal Storage Diseases, Nervous System

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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

Recruiting

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

NCT04880356

Conditions: 2-Hydroxyglutaricaciduria, Adrenal Gland Diseases, Adrenal Insufficiency, +58 more
Locations: Fondazione IRCCS Istituto Neurologico Carlo Besta Milan, Milano, Italy

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Recruiting

A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD

NCT06557369

Conditions: Body Temperature Changes, Brain Diseases, Brain Diseases, Metabolic, +24 more
Locations: Humanitas Castelli Bergamo, Italy

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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

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

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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Recruiting

Canadian Fabry Disease Initiative (CFDI) National Registry

NCT00455104

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Alberta Children's Hospital Calgary, Alberta, Canada

Vancouver General Hospital Adult Metabolic Diseases Clinic Vancouver, British Columbia, Canada

Queen Elizabeth II Health Sciences Centre Halifax, Nova Scotia, Canada

Toronto Western Hospital Toronto, Ontario, Canada

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Lysosomal Storage Diseases, Nervous System

Lysosomal Storage Diseases, Nervous System trial results

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