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Clinical Trials on Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Total 288 results
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Natera, Inc.CompletedConcurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD) (IVF008)Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)United States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedTay Sachs Disease | Sandhoff Disease | Late Onset Tay Sachs DiseaseUnited States
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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Moscow State University of Medicine and DentistryNot yet recruitingRenal Function Disorder | Simple Renal Cyst
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University of AarhusRecruitingCystic Fibrosis (CF) | CFTR Gene MutationDenmark
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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Exsar CorporationThe Hospital for Sick Children; NYU Langone Health; University Hospitals Cleveland...WithdrawnG(M2) Ganglioside | Tay-Sachs Disease Ganglioside | Sandhoff Disease GangliosideUnited States, Canada
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Idorsia Pharmaceuticals Ltd.CompletedGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
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University Hospital, MontpellierHôpital Cochin; Hôpital Necker-Enfants Malades; Foch Hospital, Suresnes, FRANCERecruitingCystic Fibrosis | CFTR Gene MutationFrance
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McGill University Health Centre/Research Institute...CompletedFamilial Dysautonomia | Tay Sachs Disease | Canavan DiseaseCanada
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Imperial College London Diabetes CentreUnknownMendelian Disorders | Genetic Disorder | Novel Mutation | Hereditary Disorder | De Novo Mutation | Inherited Disease | Single-Gene DefectsUnited Arab Emirates
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SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)RecruitingGangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator DeficiencyGermany
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Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeWithdrawnCystic Fibrosis-related Diabetes | Cystic Fibrosis Liver Disease | CF - Cystic FibrosisUnited States
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The Hospital for Sick ChildrenThe National Pancreas FoundationWithdrawnCystic Fibrosis | CFTR Gene Dysfunction
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Hospital Infantil de Mexico Federico GomezRecruitingVitamin D Deficiency | Cystic Fibrosis | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in Children | Bone Density, Low | Inflammation ChronicMexico
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Rhode Island HospitalCystic Fibrosis FoundationRecruitingCystic Fibrosis | Cystic Fibrosis-related Diabetes | Cystic Fibrosis With Intestinal ManifestationsUnited States
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Riphah International UniversityCompletedCystic Fibrosis PatientsPakistan
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Alder Hey Children's NHS Foundation TrustCompleted
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Oslo University HospitalNorwegian National Advisory Unit on Rare Disorders, Oslo University HospitalRecruitingCystic Fibrosis in ChildrenNorway
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Halic UniversityRecruitingCystic Fibrosis | Cystic Fibrosis in ChildrenTurkey
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Universidad Europea de MadridWithdrawnCystic Fibrosis in ChildrenSpain
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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University Hospital, GhentCompletedPortal Hypertension, Noncirrhotic | Cystic Fibrosis Liver Disease | Portal Hypertension Due to Cystic Fibrosis (Disorder)
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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Novartis PharmaceuticalsCompletedPulmonary Infections | Pseudomonas Aeruginosa in Cystic FibrosisUnited States, Italy, Germany, France, Australia, Canada, Spain, Hungary, Mexico, Argentina
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Federal State Budgetary Institution, Pulmonology...Moscow State University of Medicine and DentistryUnknownNon-cystic Fibrosis BronchiectasisRussian Federation
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Massachusetts General HospitalActive, not recruiting
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The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
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University of California, San FranciscoRecruitingNon-cystic Fibrosis BronchiectasisUnited States
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Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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National Human Genome Research Institute (NHGRI)RecruitingDiabetes | Undiagnosed Diseases | Inherited Metabolic Disorders | Batten's Disease | Tay SachsUnited States
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University of ReginaProvincial Health Services Authority; Saskatchewan Health Authority - Regina...Not yet recruitingMental Health | Cystic Fibrosis in Children | Siblings | Internet-based InterventionCanada
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Aradigm CorporationCompletedNon-Cystic Fibrosis BronchiectasisUnited States, Germany, Canada, United Kingdom
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Novartis PharmaceuticalsErasmus Medical Center; Fundacion Clinic per a la Recerca Biomédica; University... and other collaboratorsCompletedNon-cystic Fibrosis BronchiectasisGermany, Belgium, France, United Kingdom, Spain, Italy
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Genzyme, a Sanofi CompanyActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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Beijing Tiantan HospitalBeijing Municipal Science & Technology CommissionCompletedTransient Ischemic Attack | Stroke, Acute | Single-gene DisordersChina
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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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Assiut UniversityNot yet recruiting
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University of CambridgeUniversity of Oxford; University of EdinburghRecruitingCystic Fibrosis-related DiabetesUnited Kingdom
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Jagdeesh UllalWake Forest University Health SciencesActive, not recruitingCystic Fibrosis-related DiabetesUnited States
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Massachusetts General HospitalBeta Bionics, Inc.CompletedCystic Fibrosis-related DiabetesUnited States
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University of California, Los AngelesUniversity of Iowa; Children's Hospital Colorado; University of Kansas Medical... and other collaboratorsRecruitingChronic Rhinosinusitis (Diagnosis) | Cystic Fibrosis | Olfactory Disorder | Cystic Fibrosis in Children | Olfactory ImpairmentUnited States
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Amir MoheetRecruitingCystic Fibrosis | Cystic Fibrosis-related DiabetesUnited States
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Academisch Medisch Centrum - Universiteit van Amsterdam...RecruitingCystic Fibrosis | Cystic Fibrosis-related DiabetesNetherlands
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University Hospital, Strasbourg, FranceRecruiting
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Yale UniversityCompletedCystic Fibrosis | Cystic Fibrosis Related DiabetesUnited States
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Institut de Recherches Cliniques de MontrealVertex Pharmaceuticals Incorporated; Université de MontréalCompletedCystic Fibrosis | Cystic Fibrosis-related DiabetesCanada
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Great Ormond Street Hospital for Children NHS Foundation...University College, LondonUnknownCystic Fibrosis | Cystic Fibrosis Related DiabetesUnited Kingdom