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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

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

NCT06818838

Conditions: Anemia, Brain Diseases, Brain Diseases, Metabolic, +18 more
Locations: Guangzhou First People's Hospital Guangzhou, Guangdong, China

Shanxi Bethune Hospital Taiyuan, Shanxi, China

Hematology Hospital, Chinese Academy of Medical Sciences Tianjin, Tianjin Municipality, China

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Recruiting

Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques

NCT07336394

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +53 more
Locations: Fuwai Hospital Beijing, China

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Recruiting

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

NCT05473637

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +37 more
Locations: Department of Neurology, National Taiwan University Hospital Taipei, Taiwan

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Recruiting

Avalglucosidase Alfa Pregnancy Study

NCT05734521

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Investigational site worldwide Bridgewater, New Jersey, United States

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Recruiting

Institutional Registry of Rare Diseases

NCT06573723

Conditions: Abnormalities, Multiple, Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, +144 more
Locations: Hospital Italiano de Buenos Aires Buenos Aires, Argentina

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Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +119 more
Locations: UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States

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Recruiting

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

NCT05586243

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: University of Minnesota Minneapolis, Minnesota, United States

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Recruiting

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

NCT07187440

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: The First Affiliated Hospital Of USTC (AnHui Provincial Hospital) Hefei, Anhui, China

Peking University First Hospital Beijing, Beijing Municipality, China

Peking University Third Hospital Beijing, Beijing Municipality, China

Peking Union Medical College Hospital Beijing, Beijing Municipality, China

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Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
Locations: UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States

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Recruiting

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

NCT03952637

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations: National Institutes of Health Clinical Center Bethesda, Maryland, United States

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Lysosomal Storage Diseases, Nervous System

Lysosomal Storage Diseases, Nervous System trial results

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