Clinical Trials on Infantile GM2 Gangliosidosis (Disorder) in United Kingdom
Total 123 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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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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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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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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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
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Passage Bio, Inc.Active, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, Canada, United Kingdom, Turkey, Brazil
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Exsar CorporationThe Hospital for Sick Children; NYU Langone Health; University Hospitals Cleveland...WithdrawnG(M2) Ganglioside | Tay-Sachs Disease Ganglioside | Sandhoff Disease GangliosideUnited States, Canada
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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Massachusetts General HospitalActive, not recruiting
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University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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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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Masonic Cancer Center, University of MinnesotaCompletedWolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell DiseaseUnited States
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Masonic Cancer Center, University of MinnesotaCompletedNiemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff DiseaseUnited States
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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Genzyme, a Sanofi CompanyActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedTay Sachs Disease | Sandhoff Disease | Late Onset Tay Sachs DiseaseUnited States
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EarliTec Diagnostics, IncMarcus Autism CenterRecruitingAutism Spectrum Disorder | Developmental Delay | Autism | Autism, Early Infantile | Autism, InfantileUnited States
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National Center for Research Resources (NCRR)Icahn School of Medicine at Mount SinaiCompletedMetabolism, Inborn Errors | Leukodystrophy, Globoid Cell | Tay-Sachs Disease | Porphyria, ErythropoieticUnited States
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Yellen & Associates, Inc.WithdrawnAutistic Disorder | Asperger Syndrome | Autism, Early Infantile | Autism, InfantileUnited States
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky DiseaseUnited States, Italy, Germany, United Kingdom
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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Menarini GroupCompletedInfantile Colic | Infantile Functional Gastrointestinal DisordersUnited States
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Children's Hospital of PhiladelphiaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)RecruitingGenetic Disease | Hypercalciuria | Idiopathic Infantile Hypercalcemia - Mild Form | Idiopathic Infantile Hypercalcaemia - Severe Form | Hypercalcemia, Idiopathic, of Infancy | Hypercalciuric HypercalcemiaUnited States
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Amicus TherapeuticsCompletedVariant Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaboratorsCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedInfantile Neronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityTerminatedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityNational Institutes of Health (NIH)CompletedBatten Disease | Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Akron Children's HospitalCompletedInfantile Nystagmus SyndromeUnited States
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Jaeb Center for Health ResearchNational Eye Institute (NEI)CompletedInfantile Esotropia | Acquired Non-accommodative Esotropia | Acquired Partially Accommodative EsotropiaUnited States
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BioMarin PharmaceuticalActive, not recruitingLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2United States
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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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Children's Hospital of PhiladelphiaIllumina, Inc.Active, not recruitingMucopolysaccharidoses | 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 Gangliosidosis | Zellweger... and other conditionsUnited States
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Boston Children's HospitalRecruitingEpilepsy | Dravet Syndrome | Infantile Spasms | Epileptic Encephalopathy | Ohtahara Syndrome | Early Myoclonic Epileptic Encephalopathy | PCDH19-related Epilepsy and Related Conditions | KCNQ2-Related Epileptic Encephalopathy | Epilepsy of Infancy With Migrating Focal Seizures (Disorder)United States
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Novartis PharmaceuticalsRecruitingNLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 MutationsSpain, France, United States, Canada, Japan, United Kingdom, Turkey, Czechia, Italy
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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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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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Clinic for Special ChildrenRecruitingMyopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANMUnited States
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StemCells, Inc.CompletedNeuronal Ceroid LipofuscinosisUnited States
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St. Jude Children's Research HospitalNational Cancer Institute (NCI); Bayer/OnyxCompletedAcute Myeloid Leukemia | Myelodysplastic Syndrome | Myelodysplastic/Myeloproliferative Neoplasms | Biphenotypic Leukemia | Infantile Leukemia (Both AML and ALL)United States
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Mirum Pharmaceuticals, Inc.Children's Hospital Medical Center, CincinnatiCompletedCholestasis | Peroxisomal Disorders | Adrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's DiseaseUnited States
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University of Maryland, BaltimoreCompletedOsteoporosis PseudogliomaUnited States
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University of Maryland, BaltimoreChildren's Hospital of PhiladelphiaWithdrawnOsteoporosis Pseudoglioma SyndromeUnited States