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Clinical Trials on Purine-Pyrimidine Metabolism, Inborn Errors in New Zealand
Total 697 results
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Universität Duisburg-EssenWeinmann Geräte für Medizin GmbH + Co. KGCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy | Chronic Respiratory InsufficiencyGermany
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | Prophylaxis | HAEAustria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Switzerland, United Kingdom, United States, Australia, Hong Kong, Korea, Republic of, New Zealand, Serbia, Slovakia, South Africa, Spain
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BioMarin PharmaceuticalRecruitingHypochondroplasiaUnited States, Spain, Canada, France, Japan, Australia, Italy, Argentina, Brazil, Germany, United Kingdom
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Ultragenyx Pharmaceutical IncMereo BioPharmaCompletedOsteogenesis Imperfecta Type III | Osteogenesis Imperfecta Type IV | Osteogenesis Imperfecta, Type IUnited States, Canada, Denmark, France, United Kingdom
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ShireCompletedHereditary AngioedemaUnited States
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Natera, Inc.Children's Hospital of Philadelphia; Montefiore Medical Center; University of... and other collaboratorsCompletedPrader-Willi Syndrome | DiGeorge Syndrome | 22q11 Deletion Syndrome | Angelman Syndrome | Trisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy X | Sex Chromosome Abnormalities | 1p36 Deletion Syndrome | Cri-du-Chat SyndromeUnited States, Australia, Ireland, Spain, Sweden, United Kingdom
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ShireCompletedHereditary AngioedemaUnited States
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ShireCompletedHereditary Angioedema (HAE)United States, Jordan, Italy
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ShireCompletedHereditary AngioedemaUnited States, Australia, Israel, Canada, Hungary, Romania, Russian Federation, South Africa, Ukraine
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Grifols Therapeutics LLCCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Wiskott-Aldrich Syndrome | Agammaglobulinemia | Immunologic Deficiency SyndromeCanada, United States
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National Cancer Institute (NCI)Active, not recruitingLynch Syndrome | Stage III Colon Cancer AJCC v8 | Colon Adenocarcinoma | DNA Repair DisorderUnited States, Puerto Rico, Germany
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | HAEUnited States, Austria, Canada, Czechia, France, Germany, Hungary, North Macedonia, Romania, Spain, United Kingdom
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Washington University School of MedicineActive, not recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type IIUnited States, Canada
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Millendo Therapeutics SASTerminatedHyperphagia | Prader-Willi SyndromeUnited States, Belgium, Spain, France, Italy, United Kingdom, Australia, Netherlands
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ShireCompletedHereditary Angioedema (HAE)United States
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ShireDyax Corp.CompletedHereditary Angioedema (HAE)United States, Canada, Germany, Italy, Jordan, Puerto Rico, United Kingdom
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ShireCompletedHereditary AngioedemaUnited States
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NYU Langone HealthRecruitingParkinson Disease | Multiple System Atrophy | Neurogenic Orthostatic Hypotension | REM Sleep Behavior Disorder | Pure Autonomic Failure | Dementia With Lewy Bodies | Patients With Synucleinopathies | Shy-Drager DiseaseKorea, Republic of, United States, Argentina, Spain
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ShireCompletedHereditary Angioedema (HAE)United States, United Kingdom, Germany, Canada, Italy, Jordan, Puerto Rico
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Quince Therapeutics S.p.A.CompletedGenetic Syndrome | Nervous System DiseaseIndia, United States, Australia, Germany, Italy, Norway, Poland, Spain, Tunisia, United Kingdom, Israel, Belgium
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Alnylam PharmaceuticalsCompletedAcute Hepatic Porphyria | Acute Intermittent Porphyria | Porphyria, Acute Intermittent | Acute Porphyria | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP)United States, Spain, United Kingdom, Korea, Republic of, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Sweden, Taiwan
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ShireCompletedHereditary Angioedema (HAE)United States, Germany, Spain, Sweden
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Scholar Rock, Inc.CompletedNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type 2United States, Spain, Italy, Netherlands
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Reata, a wholly owned subsidiary of BiogenCompletedFocal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 DiabetesUnited States
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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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BioCryst PharmaceuticalsCompletedHereditary Angioedema | HAEFrance, United Kingdom, Germany, United States, Belgium, Hungary, Italy, Canada
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Clementia Pharmaceuticals Inc.TerminatedExostoses, Multiple HereditaryUnited States, Spain, Canada, Japan, Italy, Portugal, United Kingdom, Turkey, Australia, Belgium, France, Netherlands
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ShireCompletedHereditary Angioedema (HAE)Spain, Israel, United States, Hungary, Germany, Canada, Romania
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ShireCompletedHereditary Angioedema (HAE)United States, Canada, Australia, Austria, Colombia, Germany, Hungary, Israel, Italy, Spain
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TakedaTakeda Development Center Americas, Inc.CompletedAngioedemaUnited States, Spain, Netherlands, Hungary, Italy, Canada, Germany, France, Japan, Poland
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CSL BehringCompletedHereditary AngioedemaBulgaria, United States, Poland, Spain, Hungary, United Kingdom, Russian Federation, Macedonia, The Former Yugoslav Republic of, Argentina, Australia, Canada, Czech Republic, Israel, Romania, Sweden
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Scholar Rock, Inc.Active, not recruitingNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Anti-myostatin | Spinal Muscular Atrophy Type IIUnited States, Spain, Belgium, Germany, Netherlands, Poland, Italy, France, United Kingdom
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CSL BehringCompletedHereditary Angioedema Types I and IIUnited States, Spain, Germany, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
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CND Life SciencesCompletedParkinson Disease | Multiple System Atrophy | Pure Autonomic Failure | Dementia With Lewy BodiesUnited States
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CSL BehringCompletedHereditary Angioedema Types I and IIUnited States, Spain, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
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Rhythm Pharmaceuticals, Inc.CompletedBardet Biedl Syndrome (BBS) | Alström Syndrome (AS)United States, United Kingdom, France, Canada, Puerto Rico, Spain
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Bellicum PharmaceuticalsActive, not recruitingStudy of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantLymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Anemia, Aplastic | Primary Immune Deficiency Disorder | Cytopenia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child | Hemoglobinopathy in ChildrenUnited States
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ShireTakeda Development Center Americas, Inc.RecruitingHereditary Angioedema (HAE)France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden
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Global Healthy Living FoundationUniversity of Alabama at BirminghamRecruitingHeart Failure | Stroke | Epilepsy | Multiple Sclerosis | Obesity | Diabetes | Chronic Obstructive Pulmonary Disease | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Peripheral Arterial Disease | Inflammatory Bowel Diseases | Amyotrophic Lateral Sclerosis | Psoriasis | Asthma | Muscular Dystrophy | Fibromyalgia | Alzheimer... and other conditionsUnited States
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Roswell Park Cancer InstituteCompletedPrimary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditionsUnited States
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National Cancer Institute (NCI)RecruitingLynch Syndrome | Malignant Glioma | Recurrent Childhood Ependymoma | Recurrent Medulloblastoma | Refractory Ependymoma | Refractory Medulloblastoma | Recurrent Diffuse Intrinsic Pontine Glioma | Refractory Diffuse Intrinsic Pontine Glioma | Recurrent Brain Neoplasm | Refractory Brain Neoplasm | Constitutional...United States, Canada
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Scholar Rock, Inc.Active, not recruitingNeuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type 2 | Anti-myostatinUnited States, Germany, Belgium, Spain, United Kingdom, Netherlands, France, Italy, Poland
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Duke UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingHeart Failure | Pain | Pneumonia | Hypertension | Edema | Hemophilia | Insomnia | Pulmonary Arterial Hypertension | Coagulation Disorder | Asthma in Children | Down Syndrome | Attention Deficit Hyperactivity Disorder | Bronchopulmonary Dysplasia | Hypokalemia | Hyperphosphatemia | Primary Hyperaldosteronism | Adrenal Insufficiency and other conditionsUnited States, Canada
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National Cancer Institute (NCI)Active, not recruitingLynch Syndrome | Colon Mucinous Adenocarcinoma | Colon Signet Ring Cell Adenocarcinoma | Stage IIA Colon Cancer AJCC v7 | Stage IIB Colon Cancer AJCC v7 | Stage IIC Colon Cancer AJCC v7United States, Canada, Puerto Rico, Peru, South Africa
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Roswell Park Cancer InstituteNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditionsUnited States
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University of NebraskaRecruitingMelanoma | Sarcoma | Kidney Cancer | Leukemia | Breast Cancer | Laryngeal Cancer | Hypopharyngeal Cancer | Nasopharyngeal Cancer | Multiple Myeloma | Gastric Cancer | Pancreatic Cancer | Esophageal Cancer | Small Intestine Cancer | Ovarian Cancer | Lynch Syndrome | Lung Cancer | Rectal Cancer | Prostate Cancer | Gastrointestinal... and other conditionsUnited States
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Daniel BenjaminEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedHeart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditionsUnited States, Canada, United Kingdom, Israel, Singapore