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Search clinical trials for: primary mitochondrial disease (PMD)
Total 385978 results
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University Hospital, Basel, SwitzerlandRecruiting
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Joanne Kurtzberg, MDAldagenCompletedAnemia, Aplastic | MDS | Inborn Errors of Metabolism | Congenital Marrow Failure | Congenital Immunodeficiency SyndromeUnited States
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Joanne Kurtzberg, MDDuke University; Miltenyi Biotec, Inc.AvailableHematologic Malignancies | Immune Deficiencies | Inborn Errors of Metabolism DisordersUnited States
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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AldagenTerminatedMucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage DiseasesUnited States
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Khondrion BVNot yet recruitingMitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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The University of QueenslandNot yet recruitingAtaxia TelangiectasiaAustralia
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The Emmes Company, LLCUnited Mitochondrial Disease Foundation; PolG Foundation Mito AustraliaRecruitingPolG, Primary Mitochondrial MyopathyUnited States
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Semmelweis UniversityRecruitingObesity | Polycystic Ovary Syndrome | Insulin Resistance | Primary Ovarian Insufficiency | Infertility, Female | Mitochondrial AlterationHungary
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom
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IRCCS San RaffaeleAzienda Socio Sanitaria Territoriale della Valle OlonaRecruitingMultiple SclerosisItaly
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Cristina Domínguez GonzálezRecruiting
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Beijing Tiantan HospitalRecruiting
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Children's Hospital of PhiladelphiaUniversity of Pennsylvania; National Institutes of Health (NIH); United Mitochondrial...Recruiting
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Second Affiliated Hospital, School of Medicine,...Recruiting
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ErydelRecruitingAtaxia TelangiectasiaUnited States, Australia, Belgium, Germany, India, Italy, Poland, Spain, Tunisia
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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Maastricht University Medical CenterRecruiting
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Astellas Pharma IncTerminatedPrimary Mitochondrial MyopathyUnited States
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PharmasoftCompletedPrimary Open-Angle Glaucoma (POAG)Russian Federation
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Reneo Pharma LtdTerminatedPrimary Mitochondrial MyopathySpain, Australia, Belgium, Denmark, Canada, United Kingdom, France, Germany, Hungary, Italy, Netherlands, New Zealand
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Reneo Pharma LtdCompletedPrimary Mitochondrial MyopathyUnited States, Spain, Australia, France, United Kingdom, New Zealand, Denmark, Norway, Czechia, Belgium, Canada, Germany, Hungary, Italy, Netherlands
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University of California, San DiegoDysautonomia InternationalActive, not recruitingPostural Tachycardia SyndromeUnited States
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Abliva ABActive, not recruitingPrimary Mitochondrial DiseaseBelgium, United Kingdom, United States, Spain, Denmark, France
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Children's Hospital of PhiladelphiaEnrolling by invitation
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UCB BIOSCIENCES, Inc.Zogenix, Inc.Active, not recruitingThymidine Kinase 2 DeficiencyUnited States, Israel, Spain
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Zogenix MDS, Inc.Zogenix, Inc.WithdrawnThymidine Kinase 2 Deficiency
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Zogenix MDS, Inc.Zogenix, Inc.CompletedThymidine Kinase 2 DeficiencySpain, United States, Russian Federation, Turkey
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The University of QueenslandNational Health and Medical Research Council, AustraliaCompletedAtaxia TelangiectasiaAustralia
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Taysha Gene Therapies, Inc.Withdrawn
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Stealth BioTherapeutics Inc.TerminatedPrimary Mitochondrial DiseaseUnited States
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Abliva ABCompletedMitochondrial Diseases | Mitochondrial Myopathies | MELAS Syndrome | Mitochondrial Respiratory Chain DeficienciesUnited Kingdom
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University of AarhusNovo Nordisk A/SEnrolling by invitationCardiac Allograft Vasculopathy | Acute Cellular Graft Rejection | Mitochondrial Function | Primary Graft FailureDenmark
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Stealth BioTherapeutics Inc.CompletedPrimary Mitochondrial DiseaseUnited States
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Reneo Pharma LtdTerminatedPrimary Mitochondrial MyopathyUnited Kingdom
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University Hospital, Basel, SwitzerlandCompleted
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Azienda Socio Sanitaria Territoriale degli Spedali...CompletedCerebellar Ataxia | Multiple System Atrophy | Spinocerebellar Ataxias | Spinocerebellar Degenerations | Spinocerebellar Ataxia 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Ataxia, Spinocerebellar | Ataxia, Cerebellar | Ataxia With Oculomotor ApraxiaItaly
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Stealth BioTherapeutics Inc.CompletedMitochondrial MyopathyUnited States
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Medical College of WisconsinChildren's Hospital and Health System Foundation, WisconsinCompletedVomiting | MigrainesUnited States
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Stealth BioTherapeutics Inc.CompletedPrimary Mitochondrial DiseaseUnited States, Spain, Germany, Australia, Canada, Denmark, Hungary, Italy, United Kingdom
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National Institute of Allergy and Infectious Diseases...CompletedPrimary Progressive Multiple SclerosisUnited States
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Icahn School of Medicine at Mount SinaiColumbia University; New York Stem Cell Foundation Research InstituteCompletedClinically Isolated Syndrome | Relapsing-Remitting Multiple Sclerosis | Primary Progressive Multiple Sclerosis | Secondary Progressive Multiple SclerosisUnited States
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Wills EyeThomas Jefferson University; Jefferson Medical College of Thomas Jefferson...CompletedPrimary Open Angle GlaucomaUnited States
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Okuvision GmbHCompletedPrimary Open Angle Glaucoma | Retinal Vein Occlusion | Retinitis Pigmentosa | Retinal Artery Occlusion | Dry Age Related Macular Degeneration | Hereditary Macular Degeneration | Macula Off | Treated Retina Detachment | Non-Arthritic-Anterior-Ischemic Optic-Neuropathy | Hereditary Autosomal Dominant Optic... and other conditions
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Children's Hospital of PhiladelphiaIllumina, Inc.Active, not recruitingMucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditionsUnited States