- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Genetic Loci in Japan
Total 469 results
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseUnited States, Sweden, India, Netherlands, Austria, Japan, China, Serbia, France, Croatia, Australia, Portugal, Canada, Brazil, Greece, Romania, Russian Federation
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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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Bioverativ, a Sanofi companyActive, not recruitingHemophilia AUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom
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ShireTakeda Development Center Americas, Inc.CompletedAngioedemaUnited States, France, Netherlands, Spain, Hungary, Italy, Canada, Poland, Japan, Germany
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Macedonia, The Former Yugoslav Republic of, Serbia, Malaysia, Brazil, Turkey, Lithuania, Russian Federation, Italy, Japan, Poland, Puerto Rico, Taiwan
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AstraZenecaMerck Sharp & Dohme LLCActive, not recruitingNeurofibromatosis 1 | Plexiform Neurofibroma (PN)United States, Canada, France, Italy, Germany, China, United Kingdom, Spain, Japan, Russian Federation, Australia, Poland, Brazil
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Baxalta now part of ShireCompletedHemophilia AUnited States, Spain, Netherlands, Australia, Czechia, Korea, Republic of, Poland, Germany, Taiwan, Israel, Ukraine, United Kingdom, Malaysia, Japan, Austria, Switzerland, Sweden, Bulgaria, Lithuania, Romania
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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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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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Bioverativ Therapeutics Inc.CompletedHemophilia AUnited States, Canada, Italy, Australia, Austria, Belgium, Brazil, France, Germany, Hong Kong, India, Ireland, Israel, Japan, Netherlands, New Zealand, Poland, South Africa, Spain, Sweden, Switzerland, United Kingdom
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Genzyme, a Sanofi CompanyTerminatedPolycystic Kidney, Autosomal DominantBelgium, Germany, Czechia, United States, Italy, Poland, Argentina, Australia, Austria, Canada, China, Denmark, France, Israel, Japan, Korea, Republic of, Netherlands, Portugal, Romania, Spain, Taiwan, Turkey, United Kingdom
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TakedaTakeda Development Center Americas, Inc.CompletedAngioedemaUnited States, Spain, Netherlands, Hungary, Italy, Canada, Germany, France, Japan, Poland
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Novo Nordisk A/SRecruitingHaemophilia A and B With and Without InhibitorsUnited States, Spain, Italy, Estonia, Thailand, Canada, India, Malaysia, United Kingdom, Turkey, South Africa, Lebanon, Bulgaria, France, Greece, Japan, Lithuania, Norway, Poland, Romania, Russian Federation, Sweden, Algeria, North Macedonia and more
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Bioverativ Therapeutics Inc.Swedish Orphan BiovitrumCompletedSevere Hemophilia AUnited States, United Kingdom, Spain, Hong Kong, Israel, New Zealand, Sweden, Australia, Brazil, Japan, Austria, Belgium, Italy, India, Switzerland, Canada, France, Germany, South Africa
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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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Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireCompletedHemophilia AUnited States, Spain, Netherlands, Australia, Czechia, Korea, Republic of, Taiwan, Poland, Bulgaria, United Kingdom, Germany, Turkey, Israel, Malaysia, Ukraine, Russian Federation, Austria, Switzerland, Japan, Hong Kong, Lithuania, Roman... and more
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AmgenCompletedHypercholesterolemia | Familial HypercholesterolemiaJapan
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Novartis Gene TherapiesActive, not recruitingSMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type IIIUnited States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AFrance, United States, Switzerland, Malaysia, Brazil, Canada, Japan, United Kingdom, Israel, Portugal, Ukraine, Greece, Lithuania, Puerto Rico, Turkey, Italy
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PTC TherapeuticsCompletedMitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers SyndromeUnited States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan
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TakedaCompletedLennox Gastaut Syndrome (LGS)United States, China, Canada, France, Hungary, Australia, Poland, Spain, Japan, Belgium, Greece, Serbia, Germany, Italy, Latvia, Netherlands, Russian Federation, Ukraine
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Merck Sharp & Dohme LLCActive, not recruitingHypercholesterolemia | Familial HypercholesterolemiaColombia, Israel, Taiwan, United States, Argentina, China, Germany, Italy, Japan, Korea, Republic of, Mexico, South Africa, Spain, Turkey
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Novo Nordisk A/SRecruitingHaemophilia A With Inhibitors | Haemophilia ATaiwan, Canada, Spain, Netherlands, Korea, Republic of, India, United States, United Kingdom, China, Portugal, Lithuania, Turkey, South Africa, Ireland, Italy, France, Japan, Belgium, Malaysia, Poland, Austria, Bulgaria, Denmark, Germany and more
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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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Genzyme, a Sanofi CompanyRecruitingMucopolysaccharidosis I (MPS I)Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Spain, Australia, France, Thailand, Italy, Malaysia, Austria, Greece, Japan, Taiwan, Canada, Israel, Algeria, Argentina, Bulgaria, Germany, Romania
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Novo Nordisk A/SRecruitingHaemophilia A With Inhibitors | Haemophilia AUnited States, Korea, Republic of, France, Spain, Romania, Canada, Taiwan, China, Lithuania, Netherlands, India, South Africa, Russian Federation, Austria, Belgium, Czechia, Denmark, Germany, Ireland, Italy, Japan, Latvia, Malaysia, M... and more
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CSL BehringActive, not recruitingHereditary AngioedemaAustralia, Canada, United States, New Zealand, Germany, Hong Kong, Netherlands, Taiwan, Czechia, Israel, Hungary, Japan, Russian Federation, Spain
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Denmark, Austria, Spain, Japan, Brazil, Algeria, Lithuania, Portugal, Russian Federation, Serbia, Turkey, Greece, Hong Kong, Puerto Rico, Hungary, China, Poland
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Bristol-Myers SquibbCompletedMyeloid Leukemia, ChronicGermany, India, Japan, Korea, Republic of, Turkey, Chile, China, Spain, Australia, France, Poland, Mexico, Colombia, Belgium, Austria, Italy, Peru, Greece, Brazil, Czech Republic, Hungary, Russian Federation, Netherlands, Argentina, Den... and more
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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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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Spain, Germany, Switzerland, Macedonia, The Former Yugoslav Republic of, Serbia, Taiwan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Israel, Russian Federation, Turkey, Lithuania, Poland, Japan, Latvia, Puerto...
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Alexion Pharmaceuticals, Inc.TerminatedWilson DiseaseUnited States, Spain, Taiwan, France, Czechia, Hungary, Turkey, United Kingdom, Austria, Germany, Israel, Serbia, Australia, Japan, Denmark, Korea, Republic of, New Zealand, Canada, Russian Federation, Poland, Singapore, Hong Kong
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Netherlands, Spain, Switzerland, Taiwan, Turkey, United Kingdom, Croatia, Germany, Brazil, France, Japan, Australia, Hungary, Israel, Korea, Republic of, Lithuania, Portugal, Ukraine, Greece, Italy, Denmark, Norway, Canada and more
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AFrance, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, United Kingdom, Croatia, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Italy, Japan, Norway, Bulgaria
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Spain, Germany, Switzerland, Serbia, Taiwan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Japan, Denmark, Hungary, Israel, Turkey, Russian Federation, Former Serbia and Montenegro
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KalVista Pharmaceuticals, Ltd.RecruitingHereditary AngioedemaUnited States, Bulgaria, France, Greece, Israel, Netherlands, Spain, United Kingdom, Australia, Germany, South Africa, Slovakia, Austria, New Zealand, Romania, Canada, Japan
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Novartis PharmaceuticalsCompletedLymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC)United States, United Kingdom, Germany, Italy, Russian Federation, Netherlands, Japan, Canada, Poland, France, Spain
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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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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, Romania, Slovakia, Spain, United Kingdom
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PfizerWyeth is now a wholly owned subsidiary of PfizerCompletedChronic Myeloid LeukemiaFrance, United States, Spain, Belgium, Singapore, Poland, Canada, Japan, United Kingdom, Taiwan, Hungary, Germany, Argentina, Russian Federation, Turkey, Colombia, Italy, South Africa, Brazil, Chile, China, Hong Kong, India, Korea, Republic... and more
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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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KalVista Pharmaceuticals, Ltd.RecruitingHereditary AngioedemaUnited States, Bulgaria, France, Greece, Hungary, Israel, Italy, Netherlands, New Zealand, North Macedonia, Poland, Spain, United Kingdom, Australia, Germany, South Africa, Austria, Portugal, Slovakia, Romania, Canada, Saudi Arabia, Japa...
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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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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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TakedaRecruitingLennox Gastaut Syndrome (LGS) | Dravet Syndrome (DS)United States, China, Spain, France, Belgium, Australia, Brazil, Canada, Germany, Greece, Hungary, Italy, Japan, Latvia, Netherlands, Poland, Serbia, Mexico, Russian Federation, Ukraine
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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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Novartis PharmaceuticalsActive, not recruitingTuberous Sclerosis ComplexUnited States, Spain, Belgium, Taiwan, Turkey, Australia, Korea, Republic of, Thailand, France, Hungary, United Kingdom, Italy, Japan, Canada, Russian Federation, Poland, Mexico, Colombia
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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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TakedaActive, not recruitingPhiladelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ALL)United States, China, Spain, Australia, Canada, Argentina, Korea, Republic of, Brazil, Japan, Italy, Mexico, France, Greece, Poland, Russian Federation, Austria, Taiwan, Bulgaria, Finland, Turkey