Clinical Trials on Myopathies, Structural, Congenital
Total 9189 results
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Eunice Kennedy Shriver National Institute of Child...RecruitingCHILD Syndrome | Smith Lemli Opitz Syndrome | Lathosterolosis | DesmosterolosisUnited States
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Centre d'Etude des Cellules SouchesGenethonRecruitingWolfram SyndromeFrance, Spain
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Merck Sharp & Dohme LLCRecruitingVon Hippel-Lindau Disease | Pancreatic Neuroendocrine Tumor | Pheochromocytoma/Paraganglioma | Advanced Gastrointestinal Stromal Tumor | HIF-2α Mutated CancersCanada, United States, Denmark, France, Germany, Hungary, Italy, Russian Federation, Spain, Sweden, Turkey, Israel, United Kingdom, Netherlands, Australia, China, Singapore, Japan
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LLS PedAL Initiative, LLCNational Cancer Institute (NCI); Children's Oncology GroupRecruitingAcute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Myeloid Leukemia Associated With Down Syndrome | Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic Syndrome Post Cytotoxic TherapyUnited States, Canada, Australia, Puerto Rico, New Zealand
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National Medical Research Center for Therapy and...Moscow State University of Medicine and DentistryRecruitingMedication Adherence | Adherence, Medication | Treatment Adherence | Familial Hypercholesterolemia | Motivational Interviewing | Adherence, Patient | Treatment Adherence and Compliance | Patient Compliance | Adherence | Hypercholesterolemia, Familial | Patient Adherence | Hypercholesterolemia, Autosomal Dominant and other conditionsRussian Federation
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Amylyx Pharmaceuticals Inc.Active, not recruiting
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Westfälische Wilhelms-Universität MünsterICON plcUnknownPseudoxanthoma Elasticum | Generalized Arterial Calcification in Infancy | Autosomal Recessive Hypophosphatemic RicketsGermany
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Istanbul University - Cerrahpasa (IUC)Not yet recruitingUnconsciousness | Traumatic Brain Injury | Subarachnoid Hemorrhage | Coma | Infratentorial Neoplasms | Epilepsy, Temporal Lobe | Assessment, Self | Intensive Care Neurological Disorder | Arnold Chiari Malformation | Cerebrovascular Event | Postoperative Intracranial Hematoma | Postoperative Intracranial Haemorrhage and other conditionsTurkey
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Institut National de la Santé Et de la Recherche...RecruitingNeurofibromatosis Type 1 | Ichthyosis | Ectodermal Dysplasia | Albinism | Pemphigus | Mucous Membrane Pemphigoid | Inherited Epidermolysis Bullosa | Palmoplantar Keratoderma | Incontinentia PigmentiFrance
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Ospedale San RaffaeleRecruiting
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University of MiamiNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes...RecruitingAmyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Frontotemporal Dementia | Progressive Muscular Atrophy | Hereditary Spastic ParaplegiaUnited States, South Africa
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ProgenaBiomeRecruitingAutism Spectrum Disorder | Autistic Disorder | Autism | Autism, Akinetic | Autism; Atypical | Autism; Psychopathy | Autism Fragile Syndrome X | Autism With High Cognitive Abilities | Autism-Related Speech Delay | Autism, Susceptibility to, 6 | Autism Spectrum Disorder High-Functioning | Autistic Thinking | Autism... and other conditionsUnited States
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Washington University School of MedicineEnrolling by invitation
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingAcute Lymphoblastic Leukemia | Down Syndrome | Philadelphia Chromosome Positive | Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 | Childhood B Acute Lymphoblastic Leukemia | Adult B Lymphoblastic Lymphoma | Ann Arbor Stage I B Lymphoblastic Lymphoma | Ann Arbor Stage... and other conditionsUnited States, Canada, Puerto Rico, Australia, New Zealand, Switzerland, Ireland
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Reata, a wholly owned subsidiary of BiogenCompletedFocal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 DiabetesUnited States
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Peloton Therapeutics, Inc., a subsidiary of Merck...National Institutes of Health (NIH)CompletedClear Cell Renal Cell Carcinoma | Von Hippel-Lindau Disease | Clear Cell RCC | ccRCC | VHL Gene Mutation | VHL Syndrome | VHL Gene Inactivation | VHL | Von Hippel | Von Hippel's Disease | Von Hippel-Lindau Syndrome, Modifiers ofUnited States
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Augusta UniversityCompletedVascular Diseases | Body Weight Changes | Cardiovascular Abnormalities | HIV-1-infection | Body Fat Disorder | HIV-Associated Lipodystrophy Syndrome | Antiviral Drug Adverse Reaction | Abnormality of Adipose TissueUnited States
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ANRS, Emerging Infectious DiseasesUniversity Hospital, Geneva; University of Bordeaux; International Agency for... and other collaboratorsRecruitingHIV Infections | HPV - Anogenital Human Papilloma Virus InfectionBurkina Faso, Cambodia, Côte D'Ivoire
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditionsUnited States
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University College CorkNot yet recruitingGestational Diabetes | Neonatal Encephalopathy | Diastolic Dysfunction | Trisomy 21 | Transient Tachypnea of the Newborn | Small for Gestational Age at DeliveryIreland
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UNC Lineberger Comprehensive Cancer CenterRecruitingMultiple Myeloma | Amyloidosis | Smoldering Multiple Myeloma | Plasma Cell Leukemia | Cryoglobulinemia | Light Chain Deposition Disease | Castleman's Disease | Heavy Chain Deposition Disease | Polyneuropathy Organomegaly Endocrinopathy Monoclonal Gammopathy and Skin ChangesUnited States
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Brigham and Women's HospitalOregon Health and Science University; Vanderbilt University; Geisinger ClinicCompletedMyocardial Infarction | Coronary Artery Disease | Smoking | Stroke | Hypertension | Chronic Obstructive Pulmonary Disease | Atrial Fibrillation | Asthma | Tuberculosis | Sleep Apnea | Sickle Cell Disease | Hyperlipidemia | Congestive Heart FailureUnited States
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Massachusetts General HospitalCompletedFabry Disease | Mitochondrial Disease | Small Fiber Neuropathy | Ehlers Danlos SyndromeUnited States
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Hadassah Medical OrganizationUnknownDiabetes Mellitus | Iron Metabolism Disorders | Optic Atrophy | Sensorineural Hearing Loss | Platelet Dysfunction | Gastroduodenal UlcerIsrael
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Manhattan Eye, Ear & Throat HospitalGenentech, Inc.CompletedPolypoidal Choroidal Vasculopathy | Pseudoxanthoma Elasticum | Pathological Myopia | Retinal Angiomatous Proliferation | Coats' Disease | Idiopathic Retinal Telangiectasia | Multi-focal Choroiditis | Rubeosis Iridis | Von Hippel Lindau Disease | BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)United States
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Rigshospitalet, DenmarkRecruitingPolyneuropathies | Diabetic Polyneuropathy | Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | POEMS Syndrome | Multifocal Motor Neuropathy | Charcot-Marie-Tooth | hATTR Amyloidosis | Vasculitic Neuropathy | Idiopathic NeuropathyDenmark
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University of Southern DenmarkUniversity of Bern; Naestved Hospital; Nykøbing Falster County Hospital; Ankara...RecruitingPain | Wellbeing | Neurocognitive Disorders | Nausea | Vomiting | Stress | Anxiety | Satisfaction, Patient | Fasting | Temperature Change, Body | Thirst | Sedation Complication | Shivering | Agitation,Psychomotor | Hearing and Vision Loss | Sore-throat | Post Operative DeliriumDenmark
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Mayo ClinicRecruitingHematologic Neoplasms | Myeloid Malignancy | Cytopenia | Bone Marrow Failure Syndrome | Clonal Cytopenia of Undetermined Significance | Clonal Hematopoiesis of Indeterminate Potential | Low Risk Myelodysplastic Syndrome | Inherited Bone Marrow Failure Syndrome | Clonal Expansion | Hereditary Neoplastic... and other conditionsUnited States
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Jesse NodoraNational Cancer Institute (NCI)TerminatedCoronary Artery Disease | Heart Failure | Obesity | Diabetes Mellitus, Type 2 | Cancer | Chronic Kidney Diseases | Sickle Cell Disease | COPDUnited States
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Zimmer BiometCompletedTumor | Neuroma | Mandibular Fractures | Treacher Collins Syndrome | Facial Fracture | Fibrous Dysplasia | Hemifacial Microsomia | Miller Syndrome | Osteoma of Mandibular Condyle | Cleft Face | Nager SyndromeArgentina
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Atara BiotherapeuticsRecruitingLymphoproliferative Disorders | Leiomyosarcoma | Stem Cell Transplant Complications | Solid Organ Transplant Complications | Epstein-Barr Virus (EBV)-Associated Diseases | EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD) | Allogeneic Hematopoietic Cell Transplant | EBV+ Sarcomas | EBV+ Lymphoproliferative... and other conditionsUnited States, Austria, Belgium, France, Italy, Spain, United Kingdom
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University of MiamiNational Institute of Neurological Disorders and Stroke (NINDS); St. Jude Children... and other collaboratorsEnrolling by invitationAmyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Frontotemporal Dementia | Progressive Muscular Atrophy | Hereditary Spastic Paraplegia | Multisystem ProteinopathyUnited States, South Africa, Germany
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Washington University School of MedicineNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National...CompletedDiabetes Mellitus | Ataxia | Wolfram Syndrome | Optic Nerve AtrophyUnited States
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Global Healthy Living FoundationUniversity of Alabama at BirminghamRecruitingHeart Failure | Stroke | Epilepsy | Multiple Sclerosis | Obesity | Diabetes | Chronic Obstructive Pulmonary Disease | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Peripheral Arterial Disease | Inflammatory Bowel Diseases | Amyotrophic Lateral Sclerosis | Psoriasis | Asthma | Muscular Dystrophy | Fibromyalgia | Alzheimer... and other conditionsUnited States
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Inozyme PharmaNot yet recruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | ATP-Binding Cassette Subfamily C Member 6 Deficiency
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Boston Children's HospitalLymphatic Malformation InstituteRecruitingLymphangiomatosis | Kaposiform Lymphangiomatosis | Lymphatic Malformation | Generalized Lymphatic Anomaly (GLA) | Central Conducting Lymphatic Anomaly | CLOVES Syndrome | Gorham-Stout Disease ("Disappearing Bone Disease") | Blue Rubber Bleb Nevus Syndrome | Kaposiform Hemangioendothelioma/Tufted Angioma and other conditionsUnited States
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University of California, Los AngelesUniversité de MontréalWithdrawnStroke | Epilepsy | Tuberous Sclerosis | Sturge-Weber Syndrome | Focal Cortical Dysplasia | Hemimegalencephaly | Polymicrogyria | Rasmussen Encephalitis | Gliosis | Tumor, BrainUnited States, Canada
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Nemours Children's ClinicPotentials Foundation; Walking with Giants FoundationRecruitingMOPDII | RNU4atac-opathy (e.g. MOPDI/III, Lowry-Wood Syndrome, Roifman Syndrome) | Meier-Gorlin Syndrome | Saul-Wilson Syndrome | Ligase 4 Syndrome | Microcephalic Primordial DwarfismUnited States
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RFM Renal Treatment ServicesUnknownChronic Kidney Disease | Left Ventricular Hypertrophy | Renal OsteodystrophyTurkey
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University of Milano BicoccaComitato Maria Letizia Verga,ItalyRecruitingLymphoma, Non-Hodgkin | Leukemia, Myeloid, Acute | Leukemia, B-cell | Adrenoleukodystrophy | Lymphoma, Hodgkin | Thalassemia Major | Leukemia, T Cell | DrepanocytosisItaly
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Eunice Kennedy Shriver National Institute of Child...CompletedPituitary Adenoma | Cushing's Syndrome | Carney Complex | Primary Pigmented Nodular Adrenocortical Disease | Peutz-Jeghers SyndromeUnited States
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University of CincinnatiChildren's Hospital Medical Center, CincinnatiTerminatedAdrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's Disease | Bifunctional Enzyme Deficiency
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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
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Advice Pharma Group srlRecruitingDiabetes | Chronic Kidney Diseases | Tuberous Sclerosis | Albuminuria | Treatment Adherence | Arterial Hypertension | Treatment Adherence and Compliance | Nephronophthisis | Polycystic Kidney | Chronic Glomerulonephritis | Cystic Kidney Diseases | Cortical Cystic Disease | Cystic DiseasesItaly
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University of Colorado, DenverRecruitingHearing Loss | Smith-Lemli-Opitz Syndrome | Cone-Rod DystrophyUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCystic Fibrosis | Chronic Granulomatous Disease | Primary Ciliary Dyskinesia | Pseudohypoaldosteronism | Nontuberculous Mycobacterial InfectionUnited States
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RenJi HospitalPeking University First Hospital; Shanghai Zhongshan Hospital; First Affiliated... and other collaboratorsRecruitingKidney Cancer | Renal Cell Carcinoma | ALK Gene Mutation | MET Gene Mutation | HLRCC | Renal Tumor Histology | Cutaneous Leiomyoma | BAP1 Tumor Predisposition Syndrome | VHL Syndrome | Birt-Hogg-Dube Syndrome | Familial Renal Cancer | FLCN Gene Mutation | FH Gene Mutation | Cutaneous Leiomyomata With Uterine LeiomyomataChina
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National Taiwan University HospitalRecruitingStroke | Magnetic Resonance Imaging | Fabry Disease | Cerebral Small Vessel Diseases | Cadasil | HTRA1-Related Autosomal Dominant Cerebral Angiopathy | COL4A1-Related Brain Small Vessel Disease With Haemorrhage | Next-generation SequencingTaiwan
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McGill University Health Centre/Research Institute...RecruitingDiabetes Mellitus, Type 1 | Wolfram Syndrome | Neonatal Diabetes | Monogenic Diabetes | Maturity-onset Diabetes in the Young (MODY) | Wolcott-Rallison Syndrome | Mitochondrial DiabetesCanada
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Mirum Pharmaceuticals, Inc.Children's Hospital Medical Center, CincinnatiCompletedCholestasis | Peroxisomal Disorders | Adrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's DiseaseUnited States