Clinical Trials on Familial Dysautonomia
Total 1767 results
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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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University Hospital, BrestUnknownDysautonomia | Masculinity | MECP2-Related Severe Neonatal EncephalopathyFrance
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Novartis PharmaceuticalsCompletedElevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVDUnited States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
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Universidad Rey Juan CarlosRecruitingDysautonomia | Neuromodulation | Post-Acute COVID-19 SyndromeSpain
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Kecioren Education and Training HospitalCompleted
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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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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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TakedaActive, not recruitingProgressive Familial Intrahepatic Cholestasis (PFIC)Japan
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Mirum Pharmaceuticals, Inc.WithdrawnProgressive Familial Intrahepatic Cholestasis (PFIC)
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CENTOGENE GmbH RostockWithdrawnTransthyretin Amyloidosis | Transthyretin Amyloid Cardiopathy | Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyIndia, Germany
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Marmara UniversityRecruitingFamilial Mediterranean Fever | Autonomic DysfunctionTurkey
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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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University of British ColumbiaVancouver Coastal Health Research Institute; Genome British ColumbiaRecruitingAcute Coronary Syndrome | Familial Hypercholesterolemia | STEMI | NSTEMI - Non-ST Segment Elevation MI | Familial Hypercholesterolemia - Heterozygous | Familial Hypercholesterolemia Due to Genetic Defect of Apolipoprotein B | Familial Hypercholesterolemia Due to Heterozygous LDL Receptor Mutation and other conditionsCanada
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Seoul National University HospitalRecruitingFamilial Exudative VitreoretinopathiesKorea, Republic of
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The First Hospital of Hebei Medical UniversityNot yet recruitingAutonomic Nervous System Diseases
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Tulane UniversitySaul's Light FoundationRecruiting
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PfizerCompletedTransthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) | Transthyretin Cardiomyopathy (TTR-CM) | Familial Amyloid Cardiomyopathy | Senile Systemic Amyloidosis (SSA)
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac AmyloidosisUnited States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
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Novartis PharmaceuticalsCompletedFamilial Chylomicronemia Syndrome (FCS)South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
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Washington University School of MedicineIncyte CorporationNo longer available
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Virginia Polytechnic Institute and State UniversityNot yet recruiting
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University of Mississippi Medical CenterVicor Technologies, Inc.CompletedAutonomic DysfunctionUnited States
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The Cleveland ClinicCompletedAutonomic Nervous System Diseases | Mechanical VentilationUnited States
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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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Novartis PharmaceuticalsTerminatedFamilial Chylomicronemia Syndrome (FCS) (HLP Type I)South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
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Instituto Aragones de Ciencias de la SaludHospital Clinic of Barcelona; Instituto de Salud Carlos IIICompletedObesity | Weight Loss | Familial Hypercholesterolemias | Familial Combined Hypercholesterolemia
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PfizerCompletedFamilial Amyloid PolyneuropathyUnited Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden
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Centre Hospitalier Universitaire de NiceWithdrawnFamilial AmyloidosisFrance
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Assistance Publique Hopitaux De MarseilleCompletedAmyloid Neuropathies | Transthyretin AmyloidosisFrance
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Nantes University HospitalPfizerCompletedTransthyretin Amyloidosis | Familial Amyloid NeuropathyFrance
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University of Illinois at ChicagoWithdrawnVHL Gene Mutation | Polycythemia; Familial | Erythrocytosis; Familial | HIF-2alpha Erythrocytosis | PHD2 Erythrocytosis | Chuvash Erythrocytosis
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Capital Medical UniversityPeking University First Hospital; The First Affiliated Hospital of Anhui Medical... and other collaboratorsRecruitingAlzheimer Disease | Familial Alzheimer Disease (FAD)China
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PfizerCompletedFamilial Amyloid Polyneuropathy | ATTR-PNSweden, Brazil, Germany, Argentina, France, Portugal
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University Hospital, Basel, SwitzerlandCompletedMetabolic Syndrome | Familial Combined Hyperlipidemia | Familial HypertriglyceridemiaSwitzerland
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Istituto Clinico HumanitasFondazione I.R.C.C.S. Istituto Neurologico Carlo Besta; Fondazione Salvatore...CompletedAmyotrophic Lateral Sclerosis | Postural Orthostatic Tachycardia Syndrome | Pure Autonomic Failure | Anti-Cholinergic Receptors AntibodiesItaly
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University Hospital, MontpellierCompleted
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Kyoto UniversityOsaka University; Mie University; Tokushima University; Tokyo Metropolitan Geriatric... and other collaboratorsCompletedFamilial Alzheimer Disease (FAD) | PSEN1 MutationJapan
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Vanderbilt University Medical CenterTerminatedShy-Drager Syndrome | Pure Autonomic Failure | Orthostatic Hypotension, Dysautonomic | Autonomic Nervous System DisordersUnited States
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University of CalgaryInternational Collaboration on Repair DiscoveriesUnknownSpinal Cord Injuries | Autonomic Dysreflexia | Orthostatic Hypotension, DysautonomicCanada
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Institute of Health Information and Statistics...Centre of Cardiovascular and Transplantation Surgery, Czech Republic; The Central... and other collaboratorsRecruitingFamilial HypercholesterolaemiaCzechia
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Vanderbilt University Medical CenterRecruitingIdiopathic Pulmonary Fibrosis | Familial Pulmonary Fibrosis | Familial Interstitial PneumoniaUnited States
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Fundación Hipercolesterolemia FamiliarInstituto de Salud Carlos III; Fundación Centro Nacional de Investigaciones...RecruitingFamilial HypercholesterolaemiaSpain
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Kastle Therapeutics, LLCCompletedHypercholesterolemia | Heterozygous FamilialUnited States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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AmgenCompleted
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Jonathann Kuo, MDActive, not recruitingSARS-CoV2 Infection | Post-COVID-19 Syndrome | Dysautonomia | Post Acute COVID-19 Syndrome | Long COVID | Long Covid19 | COVID-19 Recurrent | Post-Acute COVID-19 | Post-Acute COVID-19 Infection | Post Acute Sequelae of COVID-19 | Dysautonomia Like Disorder | Dysautonomia Orthostatic Hypotension Syndrome | Post... and other conditionsUnited States
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First Affiliated Hospital Xi'an Jiaotong UniversityRecruitingHomozygous Familial HypercholesterolemiaChina
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Beijing Suncadia Pharmaceuticals Co., LtdNot yet recruitingHomozygous Familial Hypercholesterolemia
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Organon and CoCompletedHypercholesterolemia | Heterozygous Familial Hypercholesterolemia