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Clinical Trials on Heterozygous Familial
Total 1171 results
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Bandim Health ProjectResearch Center for Vitamins and VaccinesCompletedVaccine Adverse Reaction | Vaccine Reaction | Infant Mortality | Heterologous Immunity | Infant Morbidity | Trained ImmunityGuinea-Bissau
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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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Alnylam PharmaceuticalsApproved for marketingAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
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Eidos Therapeutics, a BridgeBio companyWithdrawnTransthyretin-Related (ATTR) Familial Amyloid PolyneuropathyUnited States
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SOM Innovation Biotech SAHospital Vall d'HebronCompleted
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University Hospital, LilleRecruitingFamilial Partial Lipodystrophy Type 2France
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AmgenCompletedHomozygous Familial Hypercholesterolemia HoFHIndia
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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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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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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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REGENXBIO Inc.National Heart, Lung, and Blood Institute (NHLBI)TerminatedHomozygous Familial Hypercholesterolemia (HoFH)United States, Canada, Italy, Netherlands
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Seoul National University HospitalRecruitingFamilial Exudative VitreoretinopathiesKorea, Republic of
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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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Washington University School of MedicineIncyte CorporationNo longer available
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Novartis PharmaceuticalsCompletedFamilial Chylomicronemia Syndrome (FCS)South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
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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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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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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, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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AmgenCompleted
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Bandim Health ProjectEnrolling by invitationMortality | Measles Vaccine | Hospital Admission | Non-specific (Heterologous) Effects of VaccinesGuinea-Bissau
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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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Arrowhead PharmaceuticalsActive, not recruitingHomozygous Familial HypercholesterolemiaAustralia, Canada, United States, South Africa
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AkesoAD Pharmaceuticals Co., Ltd.CompletedHomozygous Familial HypercholesterolemiaChina
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.TerminatedFamilial Partial LipodystrophyUnited States, Netherlands, Belgium, Brazil, Canada, Germany, Russian Federation
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Regeneron PharmaceuticalsSanofiCompletedHomozygous Familial HypercholesterolemiaUnited States, South Africa, Canada, Greece, Japan, Ukraine, Austria, Czechia, France, Germany, Italy, Taiwan, Turkey
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Australia, Austria, Canada, France, Greece, Italy, Japan, South Africa, Ukraine
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Aegerion Pharmaceuticals, Inc.WithdrawnHomozygous Familial Hypercholesterolemia
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Novartis PharmaceuticalsRecruitingHypercholesterolaemia | Familial HypercholesterolaemiaJapan
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AmgenCompletedFamilial HypercholesterolemiaCanada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...