Search clinical trials for: Lysosomal storage disease (LSD)
Total 383724 results
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Stanford UniversityRecruiting
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University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
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Amicus TherapeuticsRecruitingFabry DiseaseSpain, United States, Japan, Australia, France, Portugal
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Fondazione SISA (Societa Italiana per lo Studio...Recruiting
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National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
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Hospices Civils de LyonRecruiting
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Amicus TherapeuticsRecruitingPompe Disease (Late-onset)United States, Japan, Australia, Taiwan, Canada
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University Hospital, MontpellierRecruiting
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University Hospital, AngersRecruiting
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Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
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Wuerzburg University HospitalCharite University, Berlin, Germany; University Hospital Muenster; Health Care...RecruitingQuality of Life | Adherence, Medication | Fabry Disease | Rare DiseasesGermany
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Ultragenyx Pharmaceutical IncPRA Health SciencesRecruitingSly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
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Assistance Publique - Hôpitaux de ParisRecruiting
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University Of PerugiaRecruitingObesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic DietingItaly
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Washington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingNiemann-Pick Disease, Type CUnited States
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Wuerzburg University HospitalAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA); St George... and other collaboratorsRecruitingFabry Disease | Rare Diseases | Hypertrophic Cardiomyopathy | Fabry Disease, Cardiac VariantGermany
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingBatten Disease | Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)United States
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Cambridge University Hospitals NHS Foundation TrustMedical Research Council; National Institute for Health Research, United KingdomRecruitingGaucher Disease, Type III | Gaucher Disease, Type IUnited Kingdom
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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GOIZETBioMarin PharmaceuticalRecruiting
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Duke UniversityRecruiting
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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State University of New York at BuffaloEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruiting
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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Shenzhen Second People's HospitalGuangzhou Women and Children's Medical Center; Shenzhen UniversityRecruitingAdrenoleukodystrophy | Metachromatic LeukodystrophyChina
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Yonsei UniversityRecruitingFabry's DiseaseKorea, Republic of
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University of MinnesotaRecruitingInflammation | Oxidative Stress | Gaucher Disease Type IUnited States
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National Taiwan University HospitalRecruiting
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Joanne Kurtzberg, MDThe Marcus FoundationRecruitingMucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo MucopolysaccharidosesUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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Duke UniversityGenzyme, a Sanofi CompanyRecruitingPompe DiseaseUnited States
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Washington... and other collaboratorsRecruiting
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St. Jude Children's Research HospitalRecruitingMucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, HematologicUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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Canadian Fabry Research ConsortiumNova Scotia Health AuthorityRecruiting
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National Human Genome Research Institute (NHGRI)Recruiting
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Eunice Kennedy Shriver National Institute of Child...RecruitingNiemann-Pick Disease, Type CUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingLysosomal Storage Disease | Cholesterol MetabolismUnited States
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National Human Genome Research Institute (NHGRI)RecruitingMyoclonus | Neurological Regression | Cherry Red Spot | Brain AtrophyUnited States
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ShireTerminatedFabry DiseaseSpain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden
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National Human Genome Research Institute (NHGRI)Enrolling by invitationParkinson Disease | Gaucher Disease | Lysosomal Storage DisordersUnited States
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Wuerzburg University HospitalTakedaActive, not recruitingLysosomal Storage Diseases | Fabry Disease | Fabry Disease, Cardiac Variant | HCM - Hypertrophic Cardiomyopathy | Anderson Fabry DiseaseGermany
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Denali Therapeutics Inc.CompletedMucopolysaccharidosis IIUnited Kingdom, United States, Netherlands
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National Human Genome Research Institute (NHGRI)CompletedParkinson Disease | Gaucher DiseaseUnited States
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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Forge Biologics, IncActive, not recruiting