Clinical Trials on Congenital Myasthenic Syndromes in United States
Total 16788 results
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Alexander KolevzonCompletedPhelan-McDermid SyndromeUnited States
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Medical College of WisconsinNational Heart, Lung, and Blood Institute (NHLBI); Pediatric Heart Network; Children...Completed
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Silence Therapeutics plcWithdrawnNon-transfusion-dependent Thalassemia | Low Risk Myelodysplastic SyndromeUnited Kingdom, Bulgaria
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UCSF Benioff Children's Hospital OaklandNational Cancer Institute (NCI)CompletedNeoplastic SyndromeUnited States
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National Pediatric Neuroinflammation Organization...CompletedOpsoclonus-myoclonus SyndromeUnited States
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Quoin PharmaceuticalsRecruitingNetherton SyndromeUnited States
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Masonic Cancer Center, University of MinnesotaCompletedDiamond-Blackfan Anemia | Shwachman-Diamond Syndrome | Kostmann's NeutropeniaUnited States
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National Institutes of Health Clinical Center (CC)National Cancer Institute (NCI)CompletedKidney Cancer | Von Hippel-lindau SyndromeUnited States
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Premaitha HealthCompletedClinical Evaluation of the IONA Test for Non-invasive Pre Natal Screening in Twin Pregnancies (TWIN)Down Syndrome | Patau Syndrome | Edward's SyndromeUnited Kingdom
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SanofiTerminatedRetinitis Pigmentosa | Usher SyndromeUnited States, France
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Zynerba Pharmaceuticals, Inc.Completed22Q Deletion SyndromeUnited States, Australia
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Stanford UniversityCompletedMarfan SyndromeUnited States
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Washington University School of MedicineShwachman Diamond Syndrome Foundation; Barnes-Jewish HospitalRecruiting
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SCRI Development Innovations, LLCNovartis PharmaceuticalsTerminated
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National Pediatric Neuroinflammation Organization...Genentech, Inc.CompletedAtaxia | Myoclonus | Opsoclonus-myoclonus Syndrome | OpsoclonusUnited States
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Progenity, Inc.Obstetrix Medical GroupUnknown
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsNot yet recruitingAicardi Goutières SyndromeUnited States
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Adeline Vanderver, MDEli Lilly and CompanyActive, not recruiting
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Stealth BioTherapeutics Inc.CompletedBarth SyndromeUnited States
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Children's Hospital Medical Center, CincinnatiRecruitingHypoplastic Left Heart Syndrome (HLHS)United States, United Kingdom, Canada
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Longeveron Inc.Active, not recruiting
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National Human Genome Research Institute (NHGRI)Active, not recruiting
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Peter L GreenbergAmgenCompletedMyelodysplastic Syndromes | Myelodysplastic Syndromes (MDS) | Blood CancerUnited States
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Brian JonasNational Cancer Institute (NCI); Celgene; Pharmacyclics LLC.CompletedPreviously Treated Myelodysplastic Syndrome | Myelodysplastic Syndrome | Therapy-Related Myelodysplastic Syndrome | Secondary Myelodysplastic Syndrome | Refractory High Risk Myelodysplastic SyndromeUnited States
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsCompletedDiGeorge Anomaly | Complete DiGeorge Anomaly | Complete DiGeorge Syndrome | Complete Atypical DiGeorge Anomaly | Complete Atypical DiGeorge SyndromeUnited States
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Bausch Health Americas, Inc.TerminatedPsychosis | Velo-cardio-facial SyndromeUnited States
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsCompletedDiGeorge Syndrome | DiGeorge Anomaly | Complete DiGeorge Anomaly | Complete DiGeorge SyndromeUnited States
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State University of New York - Upstate Medical...National Institute of Mental Health (NIMH)CompletedVelocardiofacial SyndromeUnited States
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Indiana UniversityAmerican Heart Association; Purdue UniversityCompletedLong QT SyndromeUnited States
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Hospital for Special Surgery, New YorkRecruitingCRPS (Complex Regional Pain Syndromes)United States
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UConn HealthRecruitingAplasia Cutis CongenitaUnited States
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Daiichi Sankyo, Inc.CompletedNetherton SyndromeUnited States
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NorthShore University HealthSystemUnknownEKG-QT ProlongationUnited States
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Athena ZuppaCompletedHypoplastic Left Heart | Tetrology of Fallot | Heart VentricleUnited States
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Enzyvant Therapeutics GmBHNational Institute of Allergy and Infectious Diseases (NIAID); Eunice Kennedy... and other collaboratorsApproved for marketingDiGeorge Syndrome | DiGeorge Anomaly | Complete DiGeorge Anomaly | Complete DiGeorge SyndromeUnited States
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The Champ FoundationChildren's Hospital of Philadelphia; The Cleveland ClinicRecruitingPearson Syndrome | Single Large Scale Mitochondrial DNA Deletion Syndromes (SLSMDS)United States
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Le Bonheur Children's HospitalCompletedNewborn Morbidity | Gastroschisis | Bowel Obstruction | Short Bowel Syndrome | Oral AversionUnited States
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Sumitomo Pharma America, Inc.TerminatedAnemia in Myelodysplastic SyndromesUnited States
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Medical College of WisconsinCompletedPHACE SyndromeUnited States
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Children's Hospital Medical Center, CincinnatiMemorial Sloan Kettering Cancer Center; Fred Hutchinson Cancer CenterRecruitingFanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Severe Marrow FailureUnited States
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National Center for Research Resources (NCRR)Children's Hospital of PhiladelphiaUnknownAbnormalities, Multiple | DiGeorge Syndrome | Chromosome Abnormalities | Conotruncal Cardiac Defects | Shprintzen SyndromeUnited States
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Natera, Inc.CompletedTrisomy 21 | Trisomy 18 | Trisomy 13 | Sex Chromosome Abnormalities | Microdeletion SyndromesUnited States, Spain, Taiwan
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Marco CorridoreCompletedCongenital Heart Disease | Congenital Heart DefectUnited States
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Quoin PharmaceuticalsRecruitingNetherton SyndromeUnited States
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argenxQuintiles, Inc.CompletedMyasthenia GravisItaly, United States, Belgium, Canada, Netherlands, Poland, Spain, Sweden
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Innovation Research & TrainingRecruiting
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National Heart, Lung, and Blood Institute (NHLBI)Completed
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Case Comprehensive Cancer CenterRecruitingMyelodysplastic Syndrome (MDS)United States
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Weill Medical College of Cornell UniversityNovartis PharmaceuticalsCompletedMyelodysplastic Syndrome (MDS)United States
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Washington University School of MedicineCompleted