Clinical Trials on Metabolic Inborn Brain Diseases
Total 87345 results
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Passage Bio, Inc.SuspendedLeukodystrophy, Globoid CellUnited States, Brazil, Netherlands, United Kingdom, Canada, Israel
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CENTOGENE GmbH RostockWithdrawnSplenomegaly | HepatomegalyGermany, India, Sri Lanka
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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NobelpharmaCompleted
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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Assistance Publique - Hôpitaux de ParisRecruiting
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OrphalanErgomedRecruitingWilson's DiseaseSpain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
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OrphalanCompletedWilson's DiseaseUnited Kingdom
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University College, LondonUnknown
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NobelpharmaCompleted
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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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BioMarin PharmaceuticalTerminatedLate-onset Pompe Patients Untreated or Treated With rhGAASpain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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OrphalanCompletedWilson DiseaseUnited States, United Kingdom, Belgium, Brazil, Denmark, France, Germany, Italy, Poland
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Alexion Pharmaceuticals, Inc.TerminatedWilson DiseaseSpain, Korea, Republic of, France, Germany, Japan, Australia, Poland, United Kingdom
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AlexionTerminatedWilson DiseaseUnited States, France, Germany, Spain, Canada, Singapore, Poland, Denmark, Hungary, Australia, Austria, Czechia, Hong Kong, Israel, Japan, Korea, Republic of, New Zealand, Russian Federation, Serbia, Taiwan, Turkey, United Kingdom
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Fondation Ophtalmologique Adolphe de RothschildCompletedWilson DiseaseFrance
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AlexionCompletedWilson DiseaseUnited States, Germany, Austria, Poland, United Kingdom
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Vivet Therapeutics SASRecruitingWilson's DiseaseUnited States, Denmark, Germany, United Kingdom
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Second Affiliated Hospital, School of Medicine,...Recruiting
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Univar BVAptiv SolutionsCompleted
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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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Shanghai Vitalgen BioPharma Co., Ltd.RecruitingGlutaric Acidemia Type I | Glutaric Aciduria Type IChina
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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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Fondation Ophtalmologique Adolphe de RothschildRecruiting
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Second Affiliated Hospital, School of Medicine,...Recruiting
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Yale UniversityWilson Disease AssociationRecruiting
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Alexion Pharmaceuticals, Inc.CompletedWilson DiseaseUnited States, Spain, United Kingdom, New Zealand, Denmark, Russian Federation, Canada, Singapore
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AlexionCompletedWilson DiseaseUnited States
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Alexion PharmaceuticalsCompletedWilson DiseaseUnited States, Germany, Austria, Poland, United Kingdom
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CENTOGENE GmbH RostockCompletedWilson DiseasePakistan
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Yale UniversityBausch Health Americas, Inc.CompletedWilson DiseaseUnited States
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Univar BVUnknownWilson DiseaseUnited Kingdom, Germany, Greece, Italy
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The University of Texas Health Science Center,...RecruitingLeigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing EncephalomyelopathyUnited States
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ShireCompletedMetachromatic Leukodystrophy (MLD)Australia, Denmark, Japan, France, Germany
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ShireTerminatedLate Infantile Metachromatic LeukodystrophyDenmark
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National Human Genome Research Institute (NHGRI)CompletedParkinson Disease | Gaucher DiseaseUnited 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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Rigshospitalet, DenmarkDanish Research Center for Magnetic ResonanceRecruitingGlycogen Storage Disease | Pompe Disease (Late-onset) | McArdle DiseaseDenmark
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University of LausanneUniversity of Modena and Reggio EmiliaCompletedN-Acetylneuraminic Acid Storage DiseaseSwitzerland, Italy
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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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TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
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ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
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Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationCompletedLate Infantile Metachromatic LeukodystrophyFrance
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Groupe Hospitalier Diaconesses Croix Saint-SimonTerminated