Clinical Trials on Pyruvate Metabolism, Inborn Errors in France
Total 727 results
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BioMarin PharmaceuticalCompletedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV AUnited States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
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Inozyme PharmaActive, not recruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyUnited States, Canada, Germany, France, United Kingdom
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, France, Netherlands, Puerto Rico, United Kingdom
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Assistance Publique - Hôpitaux de ParisCompletedCongenital Adrenal Hyperplasia (CAH)France
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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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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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Hospices Civils de LyonCompletedClassic Congenital Adrenal HyperplasiaFrance
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Rennes University HospitalRecruitingTransthyretin Amyloidosis | Amyloidosis, HereditaryFrance
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Chiesi Farmaceutici S.p.A.Chiesi USA, Inc.AvailableFabry DiseaseUnited States
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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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Amicus TherapeuticsCompletedFabry DiseaseFrance, Austria, United States, Belgium, Japan, United Kingdom, Denmark, Brazil, Australia, Italy
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Office of Rare Diseases (ORD)UnknownAdrenal Hyperplasia, CongenitalUnited States, Brazil, France
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Horizon Pharma USA, Inc.CompletedCystinosisFrance, United States, United Kingdom, Belgium, Italy, Netherlands
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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University Hospital, BordeauxUniversity of BordeauxCompletedAmyloid Neuropathies, FamilialFrance
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Centre Hospitalier Universitaire de NīmesCompletedTransthyretin AmyloidosisFrance
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Belgium, Brazil, France, Germany, Italy, United Kingdom
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Rocket Pharmaceuticals Inc.Completed
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II;Pompe's DiseaseCzechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
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Amicus TherapeuticsTerminatedFabry DiseaseAustralia, United States, Italy, Spain, Belgium, Turkey, Brazil, Argentina, United Kingdom, Denmark, France, Canada, Egypt
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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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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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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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REGENXBIO Inc.Active, not recruitingHurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)United States, Brazil, Israel
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METAFORA biosystemsAssistance Publique - Hôpitaux de Paris; European Commission; Ministry for Health... and other collaboratorsCompletedMovement Disorders | Intellectual Disability | Seizures | Ataxia | Glut1 Deficiency Syndrome | De Vivo DiseaseFrance
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Millendo Therapeutics US, Inc.TerminatedCongenital Adrenal HyperplasiaSpain, Israel, France, Brazil, Czechia
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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Amicus TherapeuticsCompletedFabry DiseaseUnited States, Italy, Spain, Turkey, Brazil, Canada, United Kingdom, France, Poland, Australia, Egypt, Argentina, Denmark
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Idorsia Pharmaceuticals Ltd.Active, not recruitingFabry DiseaseUnited States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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Alnylam PharmaceuticalsRecruitingPrimary Hyperoxaluria Type 1France, Spain, United States, Israel, Belgium, Switzerland, Germany, United Kingdom, Italy, Canada
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Genzyme, a Sanofi CompanyRecruitingGlycogen Storage Disease Type II | Pompe DiseaseHungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
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BioMarin PharmaceuticalApproved for marketingMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, Puerto Rico
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
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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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Alnylam PharmaceuticalsCompletedPrimary Hyperoxaluria Type 1 (PH1)United States, France, United Kingdom, Switzerland, Netherlands, Israel, Germany, United Arab Emirates
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NewAmsterdam PharmaActive, not recruitingDyslipidemias | Hypercholesterolemia | Familial Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | High CholesterolUnited States, Poland, Denmark, Czechia, China, Netherlands, Japan, Georgia
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Ultragenyx Pharmaceutical IncKyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia | Hypophosphatemic RicketsUnited States, Brazil, Argentina, Canada, Chile, Colombia
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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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Centre Hospitalier Universitaire de NiceCompleted
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GenethonRecruitingCrigler-Najjar SyndromeItaly, France, Netherlands
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Institut National de la Santé Et de la Recherche...RecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IXFrance
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2United States, France, Netherlands
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Assistance Publique - Hôpitaux de ParisRecruitingAdrenoleukodystrophy | Adrenomyeloneuropathy | Metachromatic Leukodystrophy | Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaFrance
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Regeneron PharmaceuticalsTerminatedFamilial Partial Lipodystrophy | Metabolic AbnormalitiesUnited States, France, Spain, Turkey
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Centre Hospitalier Universitaire de NīmesCompletedCerebral Amyloid Angiopathy | Hypertensive ArteriopathyFrance