Clinical Trials on Phosphoglycerate Kinase (PGK) Deficiency in New Zealand
Total 15 results
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Loulou FoundationActive, not recruitingCyclin-Dependent Kinase-Like 5 Deficiency DisorderUnited States, Spain, Italy, France, Canada, Germany, United Arab Emirates
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UCB BIOSCIENCES, Inc.Zogenix, Inc.Active, not recruitingThymidine Kinase 2 DeficiencyUnited States, Israel, Spain
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Columbia UniversityUniversitat Autonoma de Barcelona; Instituto de Salud Carlos III; University... and other collaboratorsActive, not recruitingThymidine Kinase 2 Deficiency | Mitochondrial DNA Depletion Syndrome 2 Myopathic TypeUnited States
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Agios Pharmaceuticals, Inc.Active, not recruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
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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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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-DeficiencyFrance, United States, United Kingdom, Czechia
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Agios Pharmaceuticals, Inc.Active, not recruitingPyruvate Kinase DeficiencyUnited States, Canada, Netherlands, United Kingdom, France, Italy
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)France, United Kingdom, United States, Canada, Czechia
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencyFrance, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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Boston Children's HospitalAgios Pharmaceuticals, Inc.CompletedPyruvate Kinase Deficiency | Congenital Non-Spherocytic Hemolytic AnemiaUnited States, Germany, Canada, Czechia, Italy, Netherlands
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
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7 Hills Pharma, LLCNot yet recruitingMelanoma | Kidney Cancer | Renal Cell Carcinoma | Hepatocellular Carcinoma | Colorectal Cancer | NSCLC | Non Small Cell Lung Cancer | Skin Cancer | Metastasis | Advanced Solid Tumor | Advanced Cancer | Renal Cell Cancer | Hepatocellular Cancer | Mismatch Repair Deficiency | Pleural Mesothelioma | MSI-High | Anaplastic Lymphoma... and other conditionsUnited States
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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