Clinical Trials on Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive
Total 285 results
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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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Istituto Ortopedico RizzoliCompletedBethlem Myopathy | Ullrich Congenital Muscular DystrophyItaly
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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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Alkeus Pharmaceuticals, Inc.RecruitingStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
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Alkeus Pharmaceuticals, Inc.Enrolling by invitationStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
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Laval UniversityCompleted
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Otsuka Pharmaceutical Development & Commercialization...RecruitingAutosomal Recessive Polycystic Kidney (ARPKD)United States, United Kingdom, Belgium, Poland, France, Germany, Italy
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Institut National de la Santé Et de la Recherche...RecruitingLaminopathies | Emery Dreifuss Muscular Dystrophy 2 | LMNA-Related Congenital Muscular Dystrophy | Dilated Cardiomyopathy-1AFrance
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Krystal Biotech, Inc.Active, not recruitingTGM-1 Related Autosomal Recessive Congenital IchthyosisUnited States
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Mayo ClinicNational Eye Institute (NEI)CompletedRetinal Disease | Best Vitelliform Macular Dystrophy | Bestrophinopathy | Adult Onset Vitelliform Macular Dystrophy | Autosomal Dominant VitreoretinalchoroidopathyUnited States
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Ludwig-Maximilians - University of MunichCompletedInclusion Body Myositis, Sporadic | Inclusion Body Myopathy, Autosomal-recessive | Inclusion Body Myopathy, Autosomal-dominant | Congenital Cataracts, Facial Dysmorphism, And NeuropathyGermany
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Harmony Biosciences, LLCActive, not recruitingMyotonic Dystrophy 1 | Excessive Daytime SleepinessUnited States, Canada
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Kadmon Corporation, LLCCompletedPolycystic Kidney, Autosomal RecessiveUnited States
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Otsuka Pharmaceutical Development & Commercialization...RecruitingAutosomal Recessive Polycystic Kidney Disease (ARPKD)United States, Belgium, Spain, Germany, United Kingdom, Poland
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Ohio State UniversityGilead SciencesCompletedMyotonic Dystrophy 1 | Myotonia Congenita | Paramyotonia CongenitaUnited States
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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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Avidity Biosciences, Inc.CompletedMyotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert DiseaseUnited States
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Cure CMDCongenital Muscle Disease International RegistrWithdrawnLAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)United States
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Avidity Biosciences, Inc.RecruitingPhase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)Muscular Dystrophies | Muscular Dystrophy, Facioscapulohumeral | FSHD | Facio-Scapulo-Humeral Dystrophy | FMD | Facioscapulohumeral Muscular Dystrophy 1 | FSHD2 | FSHD1 | FMD2 | Fascioscapulohumeral Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy Type 1 | Fascioscapulohumeral Muscular Dystrophy... and other conditionsUnited States, United Kingdom, Canada
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Myogem Health Company, S.L.CompletedMyotonic Dystrophy 1Spain
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Bionano GenomicsColumbia University; University of Iowa; Medical College of Wisconsin; Augusta... and other collaboratorsRecruitingIntellectual Disability | Autism Spectrum Disorder | Fragile X Syndrome | Developmental Disability | Congenital Anomaly | Facioscapulohumeral Muscular Dystrophy 1United States
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Krystal Biotech, Inc.Not yet recruitingAutosomal Recessive IchthyosisUnited States
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McMaster UniversityCompletedMuscular Dystrophies | Myotonic Dystrophy 1Canada
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Institut de Myologie, FranceAssociation Française contre les Myopathies (AFM), ParisNot yet recruitingMerosin Deficient Congenital Muscular DystrophyFrance
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Prothelia, Inc.Cure CMD; The Bönnemann Laboratory, NINDS, National Institutes of Health; Oscar... and other collaboratorsCompletedMerosin Deficient Congenital Muscular DystrophyUnited States
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Assistance Publique - Hôpitaux de ParisNot yet recruitingAutosomal Recessive Congenital Ichthyosis | Epidermolytic IchthyosisFrance
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Centre Hospitalier Universitaire de NiceRecruitingFacioscapulohumeral Muscular Dystrophy 1France, Canada
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National Institute of Neurological Disorders and...TerminatedMyotonic Dystrophy Type-1 | Myotonic Dystrophy Type-2United States
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Avidity Biosciences, Inc.Active, not recruitingNervous System Diseases | Genetic Diseases, Inborn | Musculoskeletal Diseases | Muscular Diseases | Neuromuscular Diseases | Neurodegenerative Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Heredodegenerative Disorders, Nervous System | Myotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy... and other conditionsUnited States
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Centre Hospitalier Universitaire de NiceRecruitingType 1 Facioscapulohumeral Muscular DystrophyFrance
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Juan PascualNational Institute of Neurological Disorders and Stroke (NINDS)CompletedGlucose Metabolism Disorders | Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Glut1 Deficiency Syndrome 1, Autosomal Recessive | Glucose Transporter Protein Type 1 Deficiency Syndrome | Glucose Transport Defect | GLUT1DS1United States
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Fulcrum TherapeuticsActive, not recruitingFacioscapulohumeral Muscular Dystrophy 1Netherlands
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University of Texas Southwestern Medical CenterNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruitingGlucose Metabolism Disorders | Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Glut1 Deficiency Syndrome 1, Autosomal Recessive | Glucose Transporter Protein Type 1 Deficiency Syndrome | Glucose Transport Defect | GLUT1DS1United States
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Hope Biosciences Stem Cell Research FoundationHope BiosciencesNo longer availableCongenital Muscular Dystrophy Due to Lamin A/C MutationUnited States
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Ning Wang, MD., PhD.Recruiting
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Virginia Commonwealth UniversityRadboud University Medical Center; University of California, Los Angeles; University... and other collaboratorsRecruitingMyotonic Dystrophy 1United States, Netherlands, United Kingdom, New Zealand, Germany, Italy, Canada, France
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Vrije Universiteit BrusselCompletedMyotonic Dystrophy 1Belgium
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Élise DuchesneFondation du Grand défi Pierre LavoieCompletedMyotonic Dystrophy 1Canada
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IRCCS Eugenio MedeaRecruiting
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Oslo University HospitalUniversity of Oslo; Haukeland University HospitalRecruitingMyotonic Dystrophy 1Norway
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Université de SherbrookeRare Disease FoundationCompletedMyotonic Dystrophy 1Canada
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LMU KlinikumCompleted
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Cynthia GagnonFondation du Grand défi Pierre LavoieCompletedEffects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1Myotonic Dystrophy 1Canada
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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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PepGen IncRecruitingMyotonic Dystrophy 1United States, Canada
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Norwegian School of Sport SciencesUniversity of Oslo; Oslo University Hospital; University of Copenhagen; University... and other collaboratorsCompleted
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Vertex Pharmaceuticals IncorporatedRecruitingMyotonic Dystrophy Type 1 (DM1)Canada
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Dyne TherapeuticsRecruitingMyotonic Dystrophy Type 1 (DM1)United Kingdom, France, Netherlands, Italy, Germany, New Zealand
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Medical University of ViennaUnknownPolycystic Kidney, Type 1 Autosomal Dominant Disease | Polycystic Kidney, Type 2 Autosomal Dominant DiseaseAustria
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Changlin MeiThe First Affiliated Hospital with Nanjing Medical University; The First Affiliated... and other collaboratorsCompletedPolycystic Kidney, Type 1 Autosomal Dominant DiseaseChina