Search clinical trials for: lysosomal storage diseases
Total 376526 results
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SanofiApproved for marketing
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National Taiwan University HospitalSanofiCompleted
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Assistance Publique - Hôpitaux de ParisShire International GmbHCompleted
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Eunice Kennedy Shriver National Institute of Child...TerminatedNiemann-Pick Disease, Type C1United States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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LMU KlinikumCompletedHealthy | Myotonic Dystrophy Type 2 | Pompe Disease (Late-onset) | Spinal Muscular Atrophy Type 3 | Myotonic Dystrophy Type 1 (DM1) | Facioscapulohumeral Muscular Dystrophy 1 | Inclusion Body Myositis, SporadicGermany
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Duke UniversityWithdrawnPompe Disease (Late-onset)United States
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Idorsia Pharmaceuticals Ltd.CompletedFabry DiseaseUnited States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
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Ari ZimranPfizerActive, not recruitingGaucher Disease, Type 3Israel, Turkey, India
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseCanada, Russian Federation, Tunisia
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CENTOGENE GmbH RostockTerminatedDeafness | Immunodeficiency | Mental Retardation | Gingival Hypertrophy | Skeletal Abnormalities | Facial DysmorphismGermany, Switzerland
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Abeona Therapeutics, IncTerminatedMucopolysaccharidosis III-BUnited States, Germany, France
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University of UtahGenzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States
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ActelionCompleted
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Hospices Civils de LyonCompleted
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TakedaCompletedMucopolysaccharidosis (MPS)Brazil
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Abeona Therapeutics, IncTerminatedMucopolysaccharidosis Type 3 BUnited States, Germany, France, Spain
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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SanofiCompletedGaucher Disease | SplenomegalyFrance
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type IIChina
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Neurogene Inc.TerminatedLysosomal Storage Diseases | Aspartylglucosaminuria | Aspartylglucosamidase (AGA) DeficiencyUnited States
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Genzyme, a Sanofi CompanyTerminatedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Israel
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University of FloridaLacerta TherapeuticsCompletedPompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II)United States, Russian Federation, United Kingdom, Bulgaria, India, Ukraine
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisBrazil, France, Italy, United States, Germany, United Kingdom
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Turkey
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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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Institut National de la Santé Et de la Recherche...Assistance Publique - Hôpitaux de Paris; European Leukodystrophy AssociationActive, not recruitingMetachromatic LeukodystrophyFrance
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University Hospital, ToursCompletedMucopolysaccharidosesFrance
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Fondazione TelethonActive, not recruitingMucopolysaccharidosis Type VIItaly, Turkey
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Amicus TherapeuticsCompletedFabry DiseaseUnited Kingdom, United States
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Chiesi SA/NVEnrolling by invitationNephropathic CystinosisBelgium
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Germany, Turkey
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Idorsia Pharmaceuticals Ltd.CompletedGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
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Duke UniversityCompleted
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Chiesi Farmaceutici S.p.A.CromsourceCompletedAlpha-MannosidosisFrance, Denmark, Austria, Germany, Italy
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Manchester University NHS Foundation TrustNorthern Care Alliance NHS Foundation TrustCompletedFabry DiseaseUnited Kingdom
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Massachusetts General HospitalCompletedFabry Disease | Mitochondrial Disease | Small Fiber Neuropathy | Ehlers Danlos SyndromeUnited States
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Children's Hospital of Fudan UniversityFirst Affiliated Hospital, Sun Yat-Sen University; Wuhan Union Hospital, China and other collaboratorsWithdrawn
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LYSOGENECompletedMucopolysaccharidosis IIIAGermany, Brazil, United Kingdom, France, Netherlands
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LYSOGENEActive, not recruitingMucopolysaccharidosis Type IIIAGermany, United States, United Kingdom, France, Netherlands
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Hasan Kalyoncu UniversityCompleted
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IRCCS San RaffaeleFondazione TelethonActive, not recruiting
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REGENXBIO Inc.Enrolling by invitation
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Cyclo Therapeutics, Inc.CompletedNiemann-Pick Disease, Type C1Israel, Sweden, United Kingdom
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ShireCompletedFabry DiseaseCanada, United States
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Duke UniversityCompleted
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyCompletedNiemann-Pick Disease, Type C1United States