Clinical Trials on Familial Aortopathies in United States
Total 567 results
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Eidos Therapeutics, a BridgeBio companyWithdrawnTransthyretin-Related (ATTR) Familial Amyloid PolyneuropathyUnited States
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Boston UniversityNational Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...CompletedFamilial Amyloid Polyneuropathy | Familial AmyloidosisUnited States, Italy, Japan, Sweden, United Kingdom
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Washington University School of MedicineIncyte CorporationNo longer available
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Vanderbilt University Medical CenterRecruitingIdiopathic Pulmonary Fibrosis | Familial Pulmonary Fibrosis | Familial Interstitial PneumoniaUnited States
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Alnylam PharmaceuticalsCompletedATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid NeuropathiesFrance, Germany, Portugal, United Kingdom, Sweden, Spain
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Aegerion Pharmaceuticals, Inc.University of Pennsylvania; Doris Duke Charitable FoundationCompletedHomozygous Familial HypercholesterolemiaUnited States
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Novartis PharmaceuticalsActive, not recruitingFamilial Hypercholesterolemia - HomozygousGreece, Lebanon, Turkey, France, Canada, Malaysia, Netherlands, United States
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HDL TherapeuticsMedStar Heart and Vascular InstituteUnknownHomozygous Familial Hypercholesterolemia | HoFHUnited States
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National Institute of Diabetes and Digestive and...RecruitingLipodystrophyUnited States
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The Rogosin InstituteRecruitingHomozygous Familial HypercholesterolemiaUnited States
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M.D. Anderson Cancer CenterNot yet recruitingFamilial Platelet Disorder | HematopoieticUnited States
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Mayo ClinicCompleted
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Mayo ClinicUniversity of California, San Francisco; University of MinnesotaCompletedFamilial Pancreatic CancerUnited States
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REGENXBIO Inc.National Heart, Lung, and Blood Institute (NHLBI)TerminatedHomozygous Familial Hypercholesterolemia (HoFH)United States, Canada, Italy, Netherlands
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Novartis PharmaceuticalsTerminatedFamilial Chylomicronemia Syndrome (FCS) (HLP Type I)South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
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Intellia TherapeuticsRecruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisUnited Kingdom, New Zealand, Sweden
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Novartis PharmaceuticalsRecruitingHeterozygous or Homozygous Familial HypercholesterolemiaNetherlands, Israel, Hungary, Italy, Germany, Spain, United States, France, Norway, Russian Federation, South Africa, Turkey, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
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Novartis PharmaceuticalsCompletedFamilial Chylomicronemia Syndrome (FCS)South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
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Intellia TherapeuticsActive, not recruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisFrance, United Kingdom, New Zealand, Sweden
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NYU Langone HealthNot yet recruitingFamilial DysautonomiaUnited States
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NYU Langone HealthNot yet recruiting
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NYU Langone HealthCompletedFamilial DysautonomiaUnited States
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The Medicines CompanyCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Abhimanyu GargCompletedFamilial Partial LipodystrophyUnited States
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NYU Langone HealthCompletedFamilial DysautonomiaUnited States
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NYU Langone HealthCompletedFamilial DysautonomiaUnited States
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NYU Langone HealthRecruitingHereditary Sensory and Autonomic Neuropathies | Familial Dysautonomia (Riley-Day Syndrome) | Hereditary Sensory and Autonomic Neuropathy 3United States, Israel
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.CompletedFamilial Partial LipodystrophyUnited States
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REGENXBIO Inc.CompletedHomozygous Familial Hypercholesterolemia (HoFH)United States
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University of PennsylvaniaUniversity of Amsterdam; University of Cape Town; University of Witwatersrand...RecruitingHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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NYU Langone HealthWithdrawnFamilial DysautonomiaUnited States
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University of MichiganAvailable
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Canada
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Mirum Pharmaceuticals, Inc.Active, not recruitingProgressive Familial Intrahepatic Cholestasis (PFIC)United States, Argentina, France, Singapore, United Kingdom, Belgium, Turkey, Austria, Brazil, Canada, Colombia, Germany, Italy, Lebanon, Mexico, Poland
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)United States, United Kingdom, Poland, France
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Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)Italy, United States, Argentina, France, Singapore, United Kingdom, Austria, Brazil, Mexico, Lebanon, Germany, Turkey, Poland, Belgium, Canada, Colombia, Hungary
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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PfizerCompletedFamilial Amyloid PolyneuropathyUnited Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinUnited States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinNew Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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University of MichiganNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)RecruitingPregnancy Related | Obesity; FamilialUnited States
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University of Texas Southwestern Medical CenterNational Heart, Lung, and Blood Institute (NHLBI); Columbia University; The Cleveland... and other collaboratorsRecruitingAmyloidosis, Familial | Amyloidosis, Hereditary | Amyloidosis Cardiac | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin Gene MutationUnited States
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University of UtahNational Institute on Aging (NIA)CompletedDementia, FamilialUnited States
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Yale UniversityCompletedFamilial Alcoholism VulnerabilityUnited States
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Regeneron PharmaceuticalsSanofiTerminatedHeterozygous Familial Hypercholesterolemia | Non-familial HypercholesterolemiaUnited States, Bulgaria, Estonia, Russian Federation, South Africa, Ukraine
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University of MichiganCompletedNAFLD | Nonalcoholic Steatohepatitis | Familial Partial LipodystrophyUnited States
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University of Alabama at BirminghamNational Institute on Minority Health and Health Disparities (NIMHD)Active, not recruitingObesity | Obesity, Childhood | Obesity; FamilialUnited States
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Washington University School of MedicineEnrolling by invitationMotor Neuron Disease | Amyotrophic Lateral Sclerosis | Lou Gehrig Disease | Familial Amyotrophic Lateral Sclerosis | Motor Neuron Disease, FamilialUnited States