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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

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

NCT00976352

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: Shands at the University of Florida Gainesville, Florida, United States

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Completed

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

NCT01411228

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

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C) Barrio Asunción, Paraguay

Morningside Medi-Clinic Morningside, South Africa

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Completed

A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00283933

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Paris, France

London, United Kingdom

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Completed

Fabry Disease Screening in ESRD Patients in West of France

NCT02719249

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +33 more
Locations: CHU de Rennes Rennes, France

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Completed

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

NCT00446550

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Beverly Hills, California, United States

Coral Springs, Florida, United States

Decatur, Georgia, United States

Iowa City, Iowa, United States

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Completed

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Coral Springs, Florida, United States

Decatur, Georgia, United States

Boston, Massachusetts, United States

London, United Kingdom

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Completed

A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease

NCT02930655

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Investigator Site Würzburg, Germany

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Completed

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations: Univ of California San Diego School of Medicine La Jolla, California, United States

University of Florida College of Medicine Gainesville, Florida, United States

University of Kansas Medical Center Kansas City, Kansas, United States

Royal Adelaide Hospital, SA Pathology Adelaide, Adelaide, SA, Australia

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Completed

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

NCT01773954

Conditions: Age-Related Macular Degeneration, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations: Mid Atlantic Retina- Cherry Hill Cherry Hill, New Jersey, United States

Mid Atlantic Retina- Huntingdon Valley Huntingdon Valley, Pennsylvania, United States

Mid Atlantic Retina- Wills Eye Institute Philadelphia, Pennsylvania, United States

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Completed

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

NCT02208661

Conditions: Behavior, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations: Emory University Decatur, Georgia, United States

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Lysosomal Storage Diseases

Lysosomal Storage Diseases trial results

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