Clinical Trials on RYR-1 Myopathy in United States
Total 2789 results
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Armgo Pharma, Inc.National Institute of Nursing Research (NINR); National Institute of Neurological... and other collaboratorsCompletedRYR-1 MyopathyUnited States
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National Institute of Nursing Research (NINR)Not yet recruitingRyanodine Receptor 1-Related Myopathy | Ryanodine Receptor 1 Related DisordersUnited States
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Newcastle-upon-Tyne Hospitals NHS TrustRecruitingX-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-LinkedUnited Kingdom
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Myotonic Dystrophy FoundationRecruitingMyotonic Dystrophy | Myotonic Dystrophy 1 | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Steinert Disease | Dystrophia Myotonica 1 | Myotonic Dystrophy 2 | Dystrophia Myotonica | Dystrophia Myotonica 2 | Myotonia Dystrophica | Myotonic Dystrophy, Congenital | Myotonic... and other conditionsUnited States
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Clinic for Special ChildrenRecruitingMyopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANMUnited States
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Newcastle-upon-Tyne Hospitals NHS TrustRecruitingBethlem Myopathy | Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2 | Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive | Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant | Bethlem Myopathy 1, Autosomal Recessive | UCMD | BTHLM1United Kingdom
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James DowlingCanadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaboratorsRecruitingX Linked Myotubular MyopathyUnited Kingdom, Canada, United States
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University of FloridaAudentes TherapeuticsCompletedX-linked Myotubular MyopathyUnited States
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Boston Children's HospitalMuscular Dystrophy AssociationRecruitingCentronuclear Myopathy | Myotubular Myopathy | Nemaline Myopathy | Central Core Disease | Congenital Fiber Type Disproportion | Multiminicore Disease | Rigid Spine Muscular Dystrophy | Undefined Congenital MyopathyUnited States
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Cure CMDUniversity of Michigan; Congenital Muscle Disease International RegistrCompleted
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Ultragenyx Pharmaceutical IncTerminatedHereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing MyopathyCanada, United States, Bulgaria
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Texas Tech University Health Sciences CenterRecruiting
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The Emmes Company, LLCUnited Mitochondrial Disease Foundation; PolG Foundation Mito AustraliaRecruitingPolG, Primary Mitochondrial MyopathyUnited States
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Astellas Gene TherapiesCompletedX-linked Myotubular MyopathyUnited States, France, United Kingdom, Canada, Germany
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Astellas Gene TherapiesCompletedMales With X-linked Myotubular Myopathy (XLMTM)United States, United Kingdom
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Rockefeller UniversityTerminatedMyopathy (Statin Associated)United States
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Oregon Health and Science UniversityCompletedIntensive Care (ICU) MyopathyUnited States
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Ultragenyx Pharmaceutical IncTerminatedHereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing MyopathyUnited States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
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Ultragenyx Pharmaceutical IncNewcastle UniversityCompletedHereditary Inclusion Body Myopathy | GNE Myopathy | Nonaka Disease | Quadriceps Sparing Myopathy (QSM) | Distal Myopathy With Rimmed Vacuoles (DMRV)United States, Bulgaria, Canada, France, United Kingdom
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Ultragenyx Pharmaceutical IncCompletedHereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka TypeUnited States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
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University of WashingtonGrifols Biologicals, LLCWithdrawnImmune-Mediated Necrotizing MyopathyUnited States
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National Institute of Nursing Research (NINR)Completed
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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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Hartford HospitalUniversity of ConnecticutUnknown
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Astellas Gene TherapiesActive, not recruitingX-Linked Myotubular MyopathyUnited States, Canada, France, Germany
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Cure CMDUniversity of Chicago; Boston Children's Hospital; Valerion Therapeutics, LLC; Congenital...Completed
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Northwestern UniversityTerminated
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Ra PharmaceuticalsTerminatedImmune Mediated Necrotizing MyopathyUnited States, Netherlands, France, United Kingdom
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Ultragenyx Pharmaceutical IncCompletedGNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)United States, Israel
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Eduardo N. ChiniRecruitingMitochondrial MyopathyUnited States
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National Institute of Arthritis and Musculoskeletal...UnknownMitochondrial MyopathyUnited States
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Reneo Pharma LtdTerminatedPrimary Mitochondrial MyopathyUnited Kingdom
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Stealth BioTherapeutics Inc.TerminatedPrimary Mitochondrial MyopathyUnited States, Germany, Canada, Italy, United Kingdom, Denmark, Hungary
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Children's Hospital of PhiladelphiaNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMitochondrial Diseases | Mitochondrial MyopathiesUnited States
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Astellas Pharma IncActive, not recruitingPrimary Mitochondrial MyopathyUnited States
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Children's Hospital of PhiladelphiaUniversity of Pennsylvania; United Mitochondrial Disease Foundation (UMDF)Recruiting
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Ultragenyx Pharmaceutical IncCompletedHereditary Inclusion Body Myopathy (HIBM)United States
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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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Ultragenyx Pharmaceutical IncCompletedHereditary Inclusion Body Myopathy | GNE MyopathyUnited States, Israel
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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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Stealth BioTherapeutics Inc.CompletedMitochondrial MyopathyUnited States
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Valerion Therapeutics, LLCInstitut de Myologie, France; GenethonCompletedMyotubular MyopathyFrance, Belgium, United States, Canada, Germany, Italy, Spain
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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMuscular Dystrophy | Myotonic Dystrophy Type 2 | Myotonic Dystrophy Type 1 | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Myotonic Muscular DystrophyUnited States
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DynacureTerminatedCentronuclear MyopathyFrance, Netherlands, United Kingdom, Belgium, Denmark, Germany
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Reata, a wholly owned subsidiary of BiogenAbbVieCompletedMItochondrial MyopathiesUnited States, Denmark
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Eunice Kennedy Shriver National Institute of Child...Completed
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University of ManchesterKing's College Hospital NHS Trust; Northern Care Alliance NHS Foundation Trust and other collaboratorsActive, not recruitingMyositis | Idiopathic Inflammatory MyopathyUnited Kingdom
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Guy's and St Thomas' NHS Foundation TrustUniversity College, London; The Whittington Hospital NHS TrustCompleted
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Newcastle UniversityUnknownHereditary Inclusion Body Myopathy | GNE MyopathyUnited Kingdom