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Clinical Trials on Aromatic Amino Acid Decarboxylase Deficiency
Total 556 results
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HLB Cell Co., Ltd.WithdrawnUrea Cycle DisorderKorea, Republic of
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Horizon Therapeutics, LLCCompletedUrea Cycle DisorderUnited States, Canada
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Great Ormond Street Hospital for Children NHS Foundation...Orchard TherapeuticsCompletedAdenosine Deaminase Deficiency | Severe Combined Immunodeficiencies (SCID)United Kingdom
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University of California, Los AngelesGreat Ormond Street Hospital for Children NHS Foundation TrustEnrolling by invitationAdenosine Deaminase Deficiency | Severe Combined Immunodeficiency (SCID)United States, United Kingdom
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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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Fresenius KabiWithdrawnMalnutrition | Pediatric ALL | Neurocognitive Deficit | Parenteral Nutrition Associated Liver Disease | Essential Fatty Acid Deficiency
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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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BioMarin PharmaceuticalSyneos HealthRecruitingPhenylketonuria, MaternalGermany, United States, Italy
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University of AarhusRecruiting
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Johns Hopkins UniversityClark Charitable Foundation Inc.CompletedOcular Albinism (OA) | Oculocutaneous Albinism (OCA)United States
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Promethera TherapeuticsCompletedUrea Cycle Disorders | Crigler Najjar SyndromeBelgium
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Medical University of ViennaCompletedHyperemesis Gravidarum | Nausea Gravidarum | Vitamin C DeficiencyAustria
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National Human Genome Research Institute (NHGRI)RecruitingHermansky-Pudlak Syndrome (HPS)United States
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Vitaflo International, LtdUniversity College London HospitalsRecruiting
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Emma Marie Caroline SlackUniversity Children's HospitalCompletedInborn Errors of Metabolism | Propionic Aciduria | Methylmalonic Aciduria | Urea Cycle DisorderSwitzerland
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Horizon Pharma Ireland, Ltd., Dublin IrelandUcyclyd Pharma, Inc.CompletedHepatic Encephalopathy | Urea Cycle DisordersUkraine
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Katarzyna SiedzikCompletedGlucose Metabolism Disorders | Body Weight | Insulin Resistance | Lipid Metabolism Disorders | Oxidative Stress | Insulin Sensitivity | Ketosis | Ketoses, Metabolic | Body Fat Disorder | Carbohydrate Metabolism Disorder | Somatic Disorders | Immunologic Factors | Trimethylamine N-oxide | Insulin TolerancePoland
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Jasper Therapeutics, Inc.Recruiting
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States
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Uta Lichter-KoneckiMedical College of Wisconsin; Children's National Research InstituteCompletedUrea Cycle Disorders | Organic AcidemiasUnited 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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Vitaflo International, LtdRecruitingPhenylketonurias | PKU | Maple Syrup Urine Disease | Homocystinuria | Glutaric Acidemia I | Hereditary TyrosinemiaUnited Kingdom
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Bambino Gesù Hospital and Research InstituteCatholic University of the Sacred HeartCompletedGenetic Disease | Retinopathy | Methylmalonic Aciduria and Homocystinuria,Cblc TypeItaly
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Fundación Española de Hematología y HemoterapíaRecruitingGaucher Disease | Acid SphingoMyelinase DeficiencySpain
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Centre Hospitalier Universitaire DijonCentre Hospitalier Universitaire Saint PierreCompletedInfant, Premature, Diseases | Fatty Acid DeficiencyFrance
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Vanderbilt UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaboratorsCompletedHermansky Pudlak SyndromeUnited States
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Jesse RomanGenentech, Inc.UnknownInterstitial Lung Disease | Hermansky Pudlak SyndromeUnited States, Puerto Rico
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National Institute of Allergy and Infectious Diseases...WithdrawnColitis | Cytokines | Hermanski-Pudlak Syndrome | Lymphocytes | Drug EvaluationUnited States
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National Human Genome Research Institute (NHGRI)TerminatedPulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool DeficiencyUnited States
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National Human Genome Research Institute (NHGRI)RecruitingPulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)United States
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William Gahl, M.D.National Human Genome Research Institute (NHGRI)CompletedPulmonary Fibrosis | Inborn Errors of Metabolism | Albinism | Oculocutaneous Albinism | Platelet Storage Pool DeficiencyUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe DiseaseUnited States, Israel, France, United Kingdom
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Mayo ClinicRecruitingCystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase DeficiencyUnited States, Canada, Iceland, Israel
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metaX Institut fuer Diatetik GmbHGreat Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...Not yet recruitingAlkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencySpain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
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Alexion PharmaceuticalsTerminated
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Alexion PharmaceuticalsNo longer availableLysosomal Acid Lipase Deficiency
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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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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited States
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Clionsky Neuro Systems Inc.CompletedBrain Injuries | Hypoxia | Parkinson's Disease | Anemia | TDP-43 Proteinopathies | Dementia | Tauopathies | Mild Cognitive Impairment | Iron Deficiency | Vitamin B 12 Deficiency | Frontotemporal Dementia | Vascular Dementia | Alzheimer's Disease | Neurodegenerative Disorders | TBI | Lewy Body Dementia | Hyperhomocysteinemia
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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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Massachusetts General HospitalAlexion PharmaceuticalsUnknownCholesterol Ester Storage Disease | Lysosomal Acid Lipase DeficiencyUnited States
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Rush University Medical CenterActive, not recruitingBreast Cancer | Ovarian Cancer | Folic Acid DeficiencyUnited States
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Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)United States
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University of MilanRecruitingCOPD | Dyspnea | ArgininemiaItaly
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Children's National Research InstituteNational Center for Advancing Translational Sciences (NCATS)RecruitingHypoxic-Ischemic Encephalopathy | Urea Cycle Disorder | Organic Acidemia | Fatty Acid Oxidation Disorder | Maple Syrup Urine Disease | Glutaric Acidemia IUnited States