Search clinical trials for: Lysosomal Storage Diseases, Nervous System
Total 248346 results
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited States
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CENTOGENE GmbH RostockShire Human Genetic Therapies, Inc.CompletedFabry Disease | Cerebrovascular AccidentAustria, Croatia, France, Germany, Poland, Portugal, Georgia
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CENTOGENE GmbH RostockShire Human Genetic Therapies, Inc.CompletedCerebrovascular AccidentAustria, Belgium, Croatia, Finland, France, Georgia, Germany, Ireland, Italy, Malta, Poland, Portugal, Spain, United Kingdom
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CENTOGENE GmbH RostockWithdrawnStroke, Acute | Cerebrovascular Accident | Cerebral StrokeGermany
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CENTOGENE GmbH RostockCompletedFabry Disease | Anderson-Fabry Disease | Fabry´s DiseaseArgentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
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CENTOGENE GmbH RostockCompleted
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CENTOGENE GmbH RostockCompleted
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ISU Abxis Co., Ltd.CompletedGaucher DiseaseAustralia
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseFrance, Poland, United States, Russian Federation, United Kingdom
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NYU Langone HealthGenzyme, a Sanofi CompanyCompleted
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Shaare Zedek Medical CenterCompleted
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Ospedale San DonatoAmicus Therapeutics; Institute of Biomedicine and Molecular Immunology - CNRCompletedHeart Diseases | Fabry DiseaseItaly
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis (MPS)United States, Argentina, Mexico, Spain, United Kingdom
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ShireTerminatedMucopolysaccharidosis (MPS)United States, Canada, United Kingdom, Turkey, Italy, Mexico, Spain
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ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosesUnited States
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Massachusetts General HospitalGenzyme, a Sanofi Company; Scott and White Hospital & ClinicTerminatedKidney Diseases | Genetic Diseases, X-Linked | End Stage Renal Disease | ESRD | Fabry Disease | Rare DiseasesUnited States
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O & O Alpan LLCUnknownLysosomal Storage DisordersUnited States
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1United States
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LMU KlinikumGenzyme, a Sanofi CompanyCompletedPompe DiseaseGermany, Taiwan, Italy
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Lysosomal and Rare Disorders Research and Treatment...SanofiUnknown
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University Hospital MuensterAmicus TherapeuticsCompleted
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Amicus TherapeuticsCompletedOpen-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseFabry DiseaseUnited States, Austria, United Kingdom, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey
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Genzyme, a Sanofi CompanyTerminatedFabry DiseaseAustria, Brazil, France, United States, Argentina, Belgium, Canada, Finland, Hungary, Norway, Poland, Spain, Taiwan, United Kingdom
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Universidad de Zaragozafundación española para el estudio y tratamiento de la Enfermedad de Gaucher... and other collaboratorsCompleted
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Boston Children's HospitalWithdrawn
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AO GENERIUMCompletedA Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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National Niemann-Pick Disease FoundationIcahn School of Medicine at Mount Sinai; Engage Health Inc.CompletedNiemann-Pick DiseasesUnited States
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Bellicum PharmaceuticalsNo longer availableHurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage DisorderUnited States
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Duke UniversityGenzyme, a Sanofi CompanyCompleted
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyWithdrawnNiemann-Pick Disease, Type C
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Valerion Therapeutics, LLCTerminatedPompe DiseaseUnited States, United Kingdom
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University of British ColumbiaMedical University of Graz; Hospital de Clinicas de Porto AlegreUnknownMorquio B Disease | GM1 Gangliosidosis Type IIICanada
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General University Hospital, PragueCharles University, Czech Republic; Amicus Therapeutics; Klinické centrum ISCAREUnknown
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Enzyvant Therapeutics GmBHCompletedFarber Disease | Farber's Disease | Farber Lipogranulomatosis | Acid Ceramidase Deficiency | Ceramidase Deficiency | N-Laurylsphingosine Deacylase Deficiency | ASAH1 MutationUnited States, Egypt, Canada, Italy, Turkey, Argentina, Germany, India, Sweden
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseFrance, Poland, United States, Russian Federation, United Kingdom
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Baylor Research InstituteShireUnknownQuality of Life | Renal Insufficiency | Cardiac EventUnited States
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited Kingdom, Finland, United States, Italy
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedParkinson's Disease | Gaucher's DiseaseUnited States
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Rigshospitalet, DenmarkUnknownCarbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditionsDenmark
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University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
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University of California, IrvineCompletedMuscle Weakness | Pompe DiseaseUnited States
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PfizerWithdrawnType 1 Gaucher DiseaseUnited States
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Xinhua Hospital, Shanghai Jiao Tong University...UnknownNiemann-Pick Disease, Type C1China
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Shenzhen Geno-Immune Medical InstituteUnknown
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIUnited States, Canada
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University Hospital, BordeauxCompleted