Clinical Trials on Genetic Diseases, Inborn in Canada
Total 25098 results
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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The Hospital for Sick ChildrenThe Physicians' Services Incorporated FoundationCompleted
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Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireCompletedPrimary Immunodeficiency Diseases (PID)Canada
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Aegerion Pharmaceuticals, Inc.CompletedFamilial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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DS BiopharmaCompletedAtopic DermatitisUnited States, Canada, South Africa
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LEO PharmaCompletedAtopic DermatitisUnited Kingdom, Finland, Canada, Denmark
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Taysha Gene Therapies, Inc.RecruitingRett SyndromeUnited States, Canada
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GAAD Medical Research Institute Inc.Unknown
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Alnylam PharmaceuticalsRecruitingCerebral Amyloid AngiopathyCanada
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University of British ColumbiaBiogenCompletedHemophilia A, Congenital | Hemophilia B, CongenitalCanada
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SanofiRegeneron PharmaceuticalsCompletedHypercholesterolemiaSlovenia, Taiwan, Russian Federation, Canada, Netherlands, Brazil, Denmark, Mexico, Spain, Turkey
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Queen's UniversityWithdrawnCataracts | Fuch's Endothelial DystrophyCanada
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McMaster Children's HospitalUnknownEpilepsy | Metabolic Acidosis | Tubular Acidosis; RenalCanada
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St. Justine's HospitalEuro-PharmCompleted
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The Hospital for Sick ChildrenThalassemia Foundation of CanadaRecruiting
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Université de SherbrookeFRAXA Research FoundationCompleted
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FRAXA Research FoundationFragile X Research Foundation of CanadaCompleted
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University of TorontoUnknownCardiovascular Diseases | Stroke | Inflammation | Diabetes | Blood Pressure | Coronary Heart Disease | Adiposity | Cardiovascular Risk Factor | Cholesterol; LipidosisCanada
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University of British ColumbiaCompleted
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The Hospital for Sick ChildrenCanadian Hemophilia SocietyCompletedHemophilia A | Hemophilia B | Factor IX Deficiency | Factor VIII DeficiencyCanada
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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AstraZenecaCompletedHomozygous Familial Hypercholesterolemia (HoFH)Malaysia, Canada, Taiwan, Belgium, Denmark, Israel
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St. Boniface HospitalAgriculture and Agri-Food Canada; Manitoba Medical Service Foundation; Canadian...UnknownDilated CardiomyopathyCanada
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QLT Inc.CompletedRetinitis Pigmentosa (RP)Canada, Ireland
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseAustralia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Spain, Australia, United Kingdom, Italy, France, Canada, Germany, Japan, Russian Federation, Saudi Arabia, Turkey
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Regeneron PharmaceuticalsSanofiCompletedStudy to Determine the Safety and Effectiveness of Dupilumab for Treatment of Atopic Dermatitis (AD)Atopic Dermatitis (AD)United States, Canada
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The Hospital for Sick ChildrenEnrolling by invitation
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Centre de recherche du Centre hospitalier universitaire...CompletedCongenital Hypothyroidism | Autoimmune Thyroid DiseaseCanada
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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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AVROBIOTerminatedGaucher DiseaseUnited States, Canada
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Arrowhead PharmaceuticalsActive, not recruitingHomozygous Familial HypercholesterolemiaAustralia, Canada, United States, South Africa
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Amicus TherapeuticsCompletedFabry DiseaseCanada, Belgium, United States, Australia, Netherlands
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The Hospital for Sick ChildrenCompletedHypertension | Congestive CardiomyopathyCanada
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, France, United Kingdom, Canada, Netherlands, Russian Federation, Turkey, Australia
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McGill University Health Centre/Research Institute...Unknown
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AstraZenecaCompletedFamilial HypercholesterolaemiaUnited States, Netherlands, Norway, Canada, Belgium
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PepGen IncRecruitingDuchenne Muscular DystrophyCanada
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaAustralia, United States, Japan, Germany, Turkey, Italy, India, France, United Kingdom, Argentina, Canada
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REGENXBIO Inc.Active, not recruitingHomozygous Familial Hypercholesterolemia (HoFH)United States, Canada, Netherlands
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CSL BehringCompletedVon Willebrand DiseaseCanada
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Aegerion Pharmaceuticals, Inc.FDA Office of Orphan Products DevelopmentCompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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Hoffmann-La RocheCompletedAtopic DermatitisUnited States, Canada
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Louis-Nicolas Veilleux Ph.D.CompletedOsteogenesis ImperfectaCanada
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Timothy EkhlassiEnrolling by invitationPain | Dermatochalasis | Dermatochalasis of Eyelid | Dermatochalasis of Both Upper Eyelids | Dermatochalasis of Both Eyes | Dermatochalasis of Right Upper Eyelid | Dermatochalasis of Left Upper EyelidCanada
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The Hospital for Sick ChildrenCompletedHemophilia A | Hemophilia BCanada, Brazil
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Santhera PharmaceuticalsSanthera PharmaceuticalsActive, not recruitingDuchenne Muscular DystrophyCanada
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Acceleron Pharma Inc. (a wholly owned subsidiary...Terminated
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PTC TherapeuticsCompletedAniridiaUnited States, Canada