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Clinical Trials on Lysosomal Acid Lipase Deficiency
Total 340 results
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Zymenex A/SEuropean CommissionCompleted
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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Zymenex A/SEuropean CommissionUnknown
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited States
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Second Affiliated Hospital of Nanchang UniversityPeking University First HospitalUnknownHypertension | HyperhomocysteinemiaChina
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Shenzhen Ausa Pharmed Co.,LtdPeking University First Hospital; China Medical University, China; Fudan University and other collaboratorsUnknownEssential HypertensionChina
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Radboud University Medical CenterCompletedMild HyperhomocysteinemiaNetherlands
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Arcturus Therapeutics, Inc.CompletedOrnithine Transcarbamylase DeficiencyNew Zealand
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Translate Bio, Inc.WithdrawnOrnithine Transcarbamylase Deficiency
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Tufts UniversityNational Heart, Lung, and Blood Institute (NHLBI)CompletedHeart Diseases | Cardiovascular Diseases | Hyperhomocysteinemia
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Bangkok Metropolitan Administration Medical College...CompletedCardiovascular Disease | Hyperhomocysteinemia
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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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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Jasper Therapeutics, Inc.Recruiting
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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Radboud University Medical CenterCompletedEpilepsy | Ketogenic Dieting | Lactic Acid Blood Increased | GLUT1DS1 | GLUT1 Deficiency Syndrome | GLUT1DS2Netherlands
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Francisco Javier Martínez NogueraCompletedInflammatory Response | Oxygen Deficiency | Oxidative Stress | Acid-Base Imbalance | Metabolic Disturbance | Muscle Loss | Power | Electrolyte Disturbance | Change, Body Weight | Changes FattySpain
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Duke UniversityCompletedHemoglobinopathies | Thalassemia | Lysosomal Storage Disease | Immunodeficiencies | Inborn Errors of Metabolism | Sickle Cell | Non Malignant Disorders | Congenital Marrow FailuresUnited States
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CENTOGENE GmbH RostockWithdrawnLipoprotein Lipase Deficiency | Inborn Error of Lipid Metabolism | Corneal ArcusGermany, India, Sri Lanka
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Centre Hospitalier Universitaire DijonCentre Hospitalier Universitaire Saint PierreCompletedInfant, Premature, Diseases | Fatty Acid DeficiencyFrance
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Passage Bio, Inc.Active, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, Canada, United Kingdom, Turkey, Brazil
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Zymenex A/SEuropean CommissionCompletedAlpha MannosidosisCzechia, Germany, Norway, United Kingdom
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Dr. Loges & Co. GmbHBioTeSys GmbHCompletedEndothelial Dysfunction | Hyperhomocysteinemia | Hypertension Grade I, Subgroup "Borderline" (WHO)Germany
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Pamlab, Inc.Clionsky Neuro Systems Inc.UnknownMild Cognitive ImpairmentUnited States
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University of California, San FranciscoRecruitingSphingolipidoses | Enzyme DeficiencyUnited States
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University Medical Center GroningenUMC Utrecht; University of Oxford; Academisch Medisch Centrum - Universiteit... and other collaboratorsCompletedVLCAD Deficiency | Fatty Acid Oxidation DefectsNetherlands
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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Rush University Medical CenterActive, not recruitingBreast Cancer | Ovarian Cancer | Folic Acid DeficiencyUnited States
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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BioMarin PharmaceuticalQuintiles, Inc.CompletedTetrahydrobiopterin DeficiencyChina
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PTC TherapeuticsCompletedBH4 Deficiency | HyperphenylalaninemiaUnited States
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Homology Medicines, IncTerminatedPhenylketonuria | Phenylketonurias | PAH DeficiencyUnited States
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Homology Medicines, IncTerminated
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Taipei Veterans General Hospital, TaiwanBioMarin PharmaceuticalUnknownPAH Deficiency | Pku Phenylketonuria
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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Greenwood Genetic CenterCompletedGalactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler DiseaseUnited States
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LMU KlinikumSeventh Framework Programme; NBIA AllianceRecruitingNeurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditionsCanada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
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metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalCompletedPhenylketonurias | Hyperphenylalaninaemia | Tetrahydrobiopterin DeficiencyUnited Kingdom
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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Assiut UniversityCompletedCognitive Dysfunction | Hypercholesterolemia | HyperhomocysteinemiaEgypt
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Barcelona Institute for Global HealthCompletedAttention Deficit Hyperactivity Disorder | Child Behavior Problem | Fatty Acid Deficiency
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National Human Genome Research Institute (NHGRI)CompletedCancer | Myelofibrosis | Pulmonary Fibrosis | Gaucher Disease | Hermansky-Pudlak Syndrome (HPS)United States
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Orphanetics Pharma Entwicklungs GmbHTerminatedPhenylalanine Hydroxylase Deficiencies
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Central Hospital, Nancy, FranceNot yet recruitingAlzheimer Disease | Homocystinemia
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Universidade Estadual de LondrinaCompletedCardiovascular Disease | Hemodialysis | Chronic Renal Failure | Uremia | HyperhomocysteinemiaBrazil
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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain