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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

Completed

Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related Symptoms

NCT02936713

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations: Hospital Vall d'Hebron - DIGESTIVE SYSTEM RESEARCH UNIT Barcelona, Spain

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Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: University of California, San Diego Medical Center San Diego, California, United States

Yale University School of Medicine New Haven, Connecticut, United States

Emory University Medical Center Decatur, Georgia, United States

Children's Memorial Hospital Chicago, Illinois, United States

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Completed

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

NCT02293382

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Pittsburgh, Pennsylvania, United States

Porte Alegre, Brazil

Amsterdam, Netherlands

Barcelona, Spain

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Completed

Establishment of Biomarkers for Fabry Disease

NCT01165697

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Eye and Ear Institute Columbus, Ohio, United States

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Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

NCT00943111

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Tower Hematology Oncology Medical Group Beverly Hills, California, United States

UCSF MS Center San Francisco, California, United States

University of Colorado Health Science Center - Aurora Aurora, Colorado, United States

Yale University School of Medicine New Haven, Connecticut, United States

View all 34 locations

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Completed

Pregnancy and Birth Outcome in Women With Pompe Disease

NCT01556516

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: O&O Alpan Fairfax, Virginia, United States

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Completed

An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

NCT02107846

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Shaare Zedek Medical Center Jerusalem, Israel

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Completed

Bone Response to Enzyme Replacement in Gaucher's Disease

NCT00001416

Conditions: Bone Diseases, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations: National Institute of Neurological Disorders and Stroke (NINDS) Bethesda, Maryland, United States

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Completed

Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

NCT02405598

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: National Taiwan University Hospital Taipei, Taiwan

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Completed

Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth

NCT02904395

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: Not specified

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