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Clinical Trials on Pyruvate Metabolism, Inborn Errors in France
Total 727 results
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PfizerCompletedFamilial Amyloid PolyneuropathyUnited Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden
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Assistance Publique - Hôpitaux de ParisCompletedGitelman Syndrome | Potassium DepletionFrance
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Advicenne PharmaNot yet recruitingCystinuriaFrance, Belgium
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Assistance Publique - Hôpitaux de ParisCompleted
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KemPharm Denmark A/SCompletedNiemann-Pick Disease, Type CSpain, France, United Kingdom, Switzerland, Denmark, Poland, Italy, Germany
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PfizerCompletedFamilial Amyloid Polyneuropathy | ATTR-PNSweden, Brazil, Germany, Argentina, France, Portugal
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinUnited States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinNew Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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Neurocrine BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, United Kingdom, France, Greece, Italy, Spain, Belgium, Canada, Germany, Poland
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PfizerCompletedCerebral Amyloid AngiopathyCanada, United States, Netherlands, France, United Kingdom
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MedDay Pharmaceuticals SACompletedAdrenoleukodystrophy | Adrenomyeloneuropathy | AMNFrance, Germany, Spain
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ZevraDenmarkActive, not recruitingNiemann-Pick Disease, Type CUnited States, Spain, United Kingdom, Denmark, France, Germany, Italy, Poland, Switzerland
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Institut National de la Santé Et de la Recherche...RecruitingPrader-Willi Syndrome | Angelman Syndrome | Beckwith-Wiedemann Syndrome | Pseudohypoparathyroidism | Silver Russell Syndrome | Transient Neonatal Diabetes Mellitus | Temple Syndrome | Kagami-Ogata Syndrome | Familial Precocious PubertyFrance
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis II (MPS II)United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
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Assistance Publique - Hôpitaux de ParisMinistry of Health, FranceCompletedHeterozygous Carriers of Gitelman SyndromeFrance
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Assistance Publique - Hôpitaux de ParisTerminatedNephrolithiasis | Dent's DiseaseFrance
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Alnylam PharmaceuticalsCompletedATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid NeuropathiesFrance, Germany, Portugal, United Kingdom, Sweden, Spain
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Crinetics Pharmaceuticals Inc.RecruitingCongenital Adrenal Hyperplasia | Classic Congenital Adrenal HyperplasiaBrazil, India, United States, Argentina, Italy, United Kingdom
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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University Hospital, ToulouseActive, not recruitingCerebral Amyloid AngiopathyFrance
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Neurocrine BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Spain, Belgium, Austria, Bulgaria, Canada, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Serbia, Sweden, United Kingdom
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Minoryx Therapeutics, S.L.Active, not recruitingCerebral AdrenoleukodystrophySpain, Germany, France, Argentina
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, France, Germany, Italy, Netherlands, United Kingdom
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Spruce BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Australia, Brazil, Estonia, Germany, Korea, Republic of, Latvia, Lithuania, Poland, Romania, Spain, Sweden, Turkey, Canada, Italy, United Kingdom
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Assistance Publique - Hôpitaux de ParisEuroFancolenCompleted
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PfizerCompletedTransthyretin Amyloidosis | Transthyretin Gene MutationsUnited States, France, Spain, Taiwan, Israel, Korea, Republic of, Denmark, Canada, Netherlands, Japan, Sweden, Brazil, Mexico, Argentina, Portugal, Belgium, Bulgaria, Cyprus, Germany, Italy, Malaysia, Romania, Saudi Arabia, Turkey, United...
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NeuroVia, Inc.WithdrawnX-Linked AdrenoleukodystrophyAustralia, Argentina, United Kingdom, Colombia, Chile, France, Russian Federation, Ukraine
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Spruce BiosciencesTerminatedCongenital Adrenal HyperplasiaUnited States, Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Korea, Republic of, Latvia, Lithuania, Netherlands, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis | Amyloidosis, HereditaryUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
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Minoryx Therapeutics, S.L.Active, not recruitingAdrenoleukodystrophySpain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
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Institut National de la Santé Et de la Recherche...RecruitingNeurofibromatosis Type 1 | Ichthyosis | Ectodermal Dysplasia | Albinism | Pemphigus | Mucous Membrane Pemphigoid | Inherited Epidermolysis Bullosa | Palmoplantar Keratoderma | Incontinentia PigmentiFrance
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Intellia TherapeuticsActive, not recruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisFrance, United Kingdom, New Zealand, Sweden
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, Germany, Argentina, Australia, France, United Kingdom
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Akcea TherapeuticsUnited BioSource, LLCRecruitingHereditary Transthyretin Amyloidosis With PolyneuropthyUnited States, France, Greece, Italy, Spain, Canada, Germany, Portugal
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Viking Therapeutics, Inc.RecruitingAdrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)United States, France, Germany, Italy, United Kingdom
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University Hospital, BordeauxRecruitingIntellectual Disability | Cystic Fibrosis | Neurodegeneration With Brain Iron Accumulation (NBIA) | Congenital Heart Defect | Albinism | Rubinstein-Taybi Syndrome | Periventricular Nodular HeterotopiaFrance
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
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University Hospital, LilleRecruitingFamilial Partial Lipodystrophy Type 2France
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more
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Assistance Publique - Hôpitaux de ParisNot yet recruiting
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PfizerCompletedTransthyretin Amyloidosis Cardiomyopathy (ATTR-CM)Italy, Romania, France, Australia, Austria, Portugal, Slovakia, Slovenia, Spain, United Kingdom
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac AmyloidosisUnited States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
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Assistance Publique - Hôpitaux de ParisNovartisCompletedSickle Cell Disease | Thalassemia | MyelodysplasiaFrance
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University Hospital, Strasbourg, FranceRecruiting
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University Hospital, AngersCompletedPseudoxanthoma ElasticumFrance
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Novartis PharmaceuticalsCompletedTransfusional Iron Overload | β-thalassemia Major | Pediatric Rare AnemiaItaly, France
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Assistance Publique - Hôpitaux de ParisCompletedRhabdomyolysis Linked to a Hereditary Disease of MetabolismFrance