Clinical Trials on Mitochondrial Cytopathies
Total 482 results
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PTC TherapeuticsActive, not recruitingFriedreich AtaxiaUnited States
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PTC TherapeuticsEnrolling by invitationFriedreich AtaxiaUnited States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain
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PTC TherapeuticsCompletedFriedreich AtaxiaUnited States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain
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Children's Hospital of PhiladelphiaFriedreich's Ataxia Research Alliance; Stealth BioTherapeutics Inc.Active, not recruitingFriedreich AtaxiaUnited States
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Metro International Biotech, LLCChildren's Hospital of PhiladelphiaCompleted
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Santhera PharmaceuticalsCompletedFriedreich's AtaxiaUnited States
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Design TherapeuticsCompleted
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Minoryx Therapeutics, S.L.CompletedFriedreich AtaxiaSpain, Belgium, France, Germany
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Larimar Therapeutics, Inc.Clinilabs, Inc.CompletedHealthy Volunteers | Friedreich AtaxiaUnited States
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Larimar Therapeutics, Inc.Veristat, Inc.; Metrum Research Group, LLCCompletedFriedreich AtaxiaUnited States
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ShireCompletedFriedreich's AtaxiaUnited States
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National Institute of Neurological Disorders and...Completed
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University of South FloridaFriedreich's Ataxia Research Alliance; Edison Pharmaceuticals IncCompleted
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University of South FloridaUnknownFriedreich's AtaxiaUnited States
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Penwest Pharmaceuticals Co.CompletedFriedreich's AtaxiaUnited States
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Federico II UniversityCompleted
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National Institute of Neurological Disorders and...Completed
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Ultragenyx Pharmaceutical IncCompletedGNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)United States, Israel
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National Human Genome Research Institute (NHGRI)National Center for Advancing Translational Sciences (NCATS); Therapeutics...CompletedGNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)United States
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Hugo W. Moser Research Institute at Kennedy Krieger...RecruitingLeukoencephalopathies | Genetic Disease | White Matter Disease | Ataxia, Cerebellar | LBSL | Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate ElevationUnited States
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Weill Medical College of Cornell UniversityRecruitingFriedreich's AtaxiaUnited States
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Byron LamNational Eye Institute (NEI)Active, not recruitingLeber's Hereditary Optic NeuropathyUnited States
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University of PittsburghFriedreich's Ataxia Research AllianceCompleted
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CENTOGENE GmbH RostockEnrolling by invitationHereditary Diseases | FXN Gene | FRDALebanon
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PTC TherapeuticsAxio Research. LLCCompleted
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Santhera PharmaceuticalsCompletedFreidreich's AtaxiaGermany, Netherlands, France, Austria, Belgium
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Murdoch Childrens Research InstituteFriedreich's Ataxia Research AllianceCompleted
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Santhera PharmaceuticalsCompletedLeber's Hereditary Optic NeuropathyCanada, Germany, United Kingdom
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LMU KlinikumSeventh Framework Programme; NBIA AllianceRecruitingNeurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditionsCanada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
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Friedreich's Ataxia Research AllianceChildren's Hospital of Philadelphia; University of RochesterRecruitingFriedreich Ataxia | Neuro-Degenerative DiseaseUnited States, Canada, New Zealand, Australia, India
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Santhera PharmaceuticalsNo longer availableLeber's Hereditary Optic Neuropathy
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University of MinnesotaFriedreich's Ataxia Research Alliance; Bob Allison Ataxia Research CenterCompletedFriedreich AtaxiaUnited States
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Taysha Gene Therapies, Inc.Withdrawn
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Stealth BioTherapeutics Inc.CompletedLeber's Hereditary Optic NeuropathyUnited States
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Adverum Biotechnologies, Inc.Adverum Biotechnologies SAS, a wholly owned subsidiary of Adverum Biotechnologies...CompletedFriedreich's AtaxiaFrance
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Benha UniversityCompletedDuchenne Muscular Dystrophy | Inflammatory MyopathyEgypt
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German Center for Neurodegenerative Diseases (DZNE)McMaster University; Sorbonne UniversityRecruitingFriedreich AtaxiaAustria, Germany, France
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PTC TherapeuticsCompletedLeigh SyndromeUnited States
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Aadi Bioscience, Inc.Withdrawn
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Santhera PharmaceuticalsWithdrawnLeber's Hereditary Optic Neuropathy
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer availableBarth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditionsUnited States
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Massachusetts General HospitalCompletedFabry Disease | Mitochondrial Disease | Small Fiber Neuropathy | Ehlers Danlos SyndromeUnited States
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University Hospital, AngersNot yet recruitingNicotinamide Adverse Reaction
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Neurophth Therapeutics IncRecruitingLeber Hereditary Optic Neuropathy (LHON)China, United States
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Wuhan Neurophth Biotechnology Limited CompanyRecruitingLeber's Hereditary Optic Neuropathy (LHON)China
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Hôpital Necker-Enfants MaladesEuropean Georges Pompidou Hospital; CLAIROPCompletedSafety Issues | Efficacy, SelfFrance
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Edison Pharmaceuticals IncNo longer availableLeber's Hereditary Optic NeuropathyUnited States
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Medical College of WisconsinTerminatedLeber's Hereditary Optic Neuropathy (LHON)United States
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University Hospital, AngersUnknownLeber Hereditary Optic NeuropathyFrance
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National Eye Institute (NEI)CompletedMacular Degeneration | Diabetic Retinopathy | Mitochondria | Hippel-Landau DiseaseUnited States