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Clinical Trials on Hurler Syndrome (MPS I)
Total 799 results
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National Cancer Institute (NCI)CompletedMDS | Immunodeficiency | GATA2United States
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BioMarin PharmaceuticalApproved for marketingMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, Puerto Rico
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Verinata Health, Inc.CompletedPregnancy | Down Syndrome | Edwards Syndrome | Patau Syndrome | Turners SyndromeUnited States
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Sunnybrook Health Sciences CentreQueen's University; Ontario Molecular Pathology Research NetworkUnknownCardiovascular Diseases | Inflammation | MDSCanada
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National Heart, Lung, and Blood Institute (NHLBI)Children's National Research InstituteCompletedLymphoma, Non-Hodgkin | MDS (Myelodysplastic Syndrome) | Myeloproliferative Disorder | ALL (Acute B-Lymphoblastic Leukemia) | AML (Acute Myelogenous LeukemiaUnited States
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.CompletedFamilial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1United States, Canada, Germany, Israel, Italy, South Africa, Spain, United Kingdom, Brazil, France, Netherlands
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.CompletedFamilial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1Canada
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Jazz PharmaceuticalsCompletedAcute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS)United States
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National Institute of Neurological Disorders and...Recruiting
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Jazz PharmaceuticalsWithdrawnAcute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS)United States
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University of Kansas Medical CenterCompletedAcute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Non-Hodgkins Lymphoma | Myelodysplastic Syndrome (MDS) | Hodgkins LymphomaUnited States
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Second Affiliated Hospital, School of Medicine,...Shanghai Zhongshan Hospital; Sir Run Run Shaw Hospital; Zhejiang Provincial People... and other collaboratorsRecruiting
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JOTEC GmbHRecruitingAortic Dissection | Intramural Hematoma | Acute DeBakey I Dissection | Acute Type A DissectionGermany
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Stichting Achmea Slachtoffer en SamenlevingCompletedRSD (Reflex Sympathetic Dystrophy) | Algodystrophy | CRPS Type INetherlands
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Children's Hospital Medical Center, CincinnatiRecruitingLow Back Pain | Chronic Widespread Pain | Fibromyalgia | Complex Regional Pain Syndrome (CRPS) | Ehlers-Danlos Syndrome (EDS) | Amplified Musculoskeletal Pain Syndrome (AMPS)United States
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Emory UniversityBioMarin PharmaceuticalCompletedMPS - Mucopolysaccharidosis | Morquio DiseaseUnited States
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Memorial Sloan Kettering Cancer CenterPediatric Brain Tumor ConsortiumTerminatedFanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML)United States
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Alexion PharmaceuticalsTerminatedMPS IIIB (Sanfilippo B Syndrome)Spain, United States, Brazil, United Kingdom, Portugal, Italy
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Arrowhead PharmaceuticalsActive, not recruitingFamilial ChylomicronemiaKorea, Republic of, Canada, Australia, Singapore, Belgium, Spain, Japan, United States, Austria, Germany, Ireland, Oman, Poland, Israel, New Zealand, Argentina, France, Croatia, Mexico, Serbia, Turkey
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Weill Medical College of Cornell UniversityActive, not recruitingMalignant Neoplasm of Breast Stage IUnited States
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Akcea TherapeuticsCaligorRx, Inc.No longer availableFamilial Chylomicronemia
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University of Novi SadMerck Sharp & Dohme LLCRecruitingMetabolic Syndrome | Aging | HIV I Infection | HIV Associated LipodystrophySerbia
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HAE Global Registry FoundationRecruitingHereditary Angioedema Type I and IIItaly
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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
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Prothya BiosolutionsCompletedHereditary Angioedema Type I | Angioneurotic EdemaNetherlands
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Sequenom, Inc.CompletedTurner Syndrome | Down Syndrome (Trisomy 21) | Patau Syndrome (Trisomy 13) | Edwards Syndrome (Trisomy 18)United States
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National Institute of Allergy and Infectious Diseases...Autoimmunity Centers of Excellence; Clinical Trials in Organ Transplantation...CompletedSevere Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) | Coronavirus Disease 2019 (COVID-19) | Multisystem Inflammatory Syndrome in Children (MIS-C)United States
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Progenity, Inc.UnknownDown Syndrome | DiGeorge Syndrome | Turner Syndrome | Klinefelter Syndrome | Edwards Syndrome | Patau Syndrome | Perinatal InfectionsUnited States
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KalVista Pharmaceuticals, Ltd.TerminatedAngioedema, Hereditary, Types I and IIUnited States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico
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Zachariah Michael DeFilippRegimmune CorporationActive, not recruitingAcute Myeloid Leukemia | Myelodysplastic Syndromes | Hodgkin Lymphoma | Chronic Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | AML | Non Hodgkin Lymphoma | MDS | Myeloproliferative Disorder | GVHD | Graft Vs Host Disease | ALL | CMML | Blood Stem Cell Transplant Failure | MPN | Chemosensitive Hodgkin LymphomaUnited States
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University of Colorado, DenverCompletedMarfan Syndrome | Turner Syndrome | Loeys-Dietz Syndrome | Thoracic Aortic Aneurysm and Dissection Syndromes | Ehlers-Danlos Type IV SyndromeUnited States
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CENTOGENE GmbH RostockTerminatedMucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal SyndromeMexico
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Radboud University Medical CenterPrincess Beatrix Muscle FoundationCompletedParsonage Turner Syndrome | Neuralgic Amyotrophy | Neuralgic Amyotrophy, Hereditary | Brachial NeuritisNetherlands
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Medical College of WisconsinChildren's Hospital of PhiladelphiaCompletedMalignant Diseases (ie, Leukemia, MDS, Lymphoma) | Non-malignant Diseases (ie, Bone Marrow Failure Syndromes)United States
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Eunice Kennedy Shriver National Institute of Child...CompletedGonadal Dysgenesis | Turner's SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedGonadal Dysgenesis | Turner's SyndromeUnited States
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National Institute of Neurological Disorders and...Jefferson Medical College of Thomas Jefferson UniversityCompletedTurner's SyndromeUnited States
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MacroGenicsNational Institute of Allergy and Infectious Diseases (NIAID); National Institutes... and other collaboratorsActive, not recruitingHuman Immunodeficiency Virus I Infection | Immunodeficiency Virus Type 1, Human | Human Immunodeficiency Virus Type 1United States
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bioLytical LaboratoriesNot yet recruitingHuman Immunodeficiency Virus I Infection | Human Immunodeficiency Virus II Infection
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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Dong-A ST Co., Ltd.Completed
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Charite University, Berlin, GermanyUnknownMultiple Sclerosis, MS | Clinically Isolated SyndromeGermany
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University of AarhusLundbeck Foundation; AP Moeller Foundation; The Korning Foundation DenmarkCompletedAortic Aneurysm | Turner Syndrome | Klinefelter Syndrome | Triple X Syndrome | 47 XYY SyndromeDenmark
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BioMarin PharmaceuticalICON plcCompletedMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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Arrowhead PharmaceuticalsCompletedHypertriglyceridemia | Familial ChylomicronemiaAustralia, Canada, New Zealand
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University of ChicagoJohns Hopkins University; Massachusetts General Hospital; Novo Nordisk A/S; University... and other collaboratorsTerminated
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National Institute of Neurological Disorders and...Jefferson Medical College of Thomas Jefferson UniversityCompletedTurner's SyndromeUnited States
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Ginna LaportTerminatedAcute Myelogenous Leukemia (AML) - Relapsed, Primary Refractory Disease or Poor Risk Factors | Chronic Myelogenous Leukemia (CML) - Accelerated or Second Chronic Phase | Myelodysplastic Syndrome (MDS) - High and Intermediate Risk | Non-Hodgkin's Lymphoma (NHL) | Chronic Lymphocytic Leukemia...United States
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Mesoblast International SàrlAvailableMultisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)