Not applicableStudy completedNCT01474980What this trial is testingPregnancy Outcomes in Congenital Myasthenie SyndromeWho this might be right forCongenital Myasthenic Syndrome Institut de Myologie, France 25
Not applicableLooking for participantsNCT06630650What this trial is testingA Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic SyndromesWho this might be right forMyasthenic Syndromes, Congenital National Institute of Neurological Disorders and Stroke (NINDS) 75
Not applicableEnrolling By InvitationNCT07072676What this trial is testingThe Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Who this might be right forInclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more LMU Klinikum 30
Not applicableLooking for participantsNCT06078553What this trial is testingA Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4Who this might be right forCongenital Myasthenic Syndrome argenx 100
Large-scale testing (Phase 3)Study completedNCT02562066What this trial is testingAmifampridine Phosphate for the Treatment of Congenital Myasthenic SyndromesWho this might be right forMyasthenic Syndromes, Congenital Catalyst Pharmaceuticals, Inc. 20
Not applicableLooking for participantsNCT07136844What this trial is testingGait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic PathologyWho this might be right forNeuromuscular DiseasesObesity (Disorder)Myotonic Dystrophy 1+9 more Centre Hospitalier Universitaire de Liege 300
Not applicableNo Longer AvailableNCT02012933What this trial is testing3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)Who this might be right forLambert-Eaton Myasthenic Syndrome (LEMS)Congenital Myasthenia (CM) Oregon Health and Science University
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000
Not applicableUnknownNCT02124616What this trial is testingNational Registry for Egyptian Pediatric Neuromuscular DiseasesWho this might be right forSpinal Muscular AtrophyMuscular DystrophyMuscle Diseases+2 more Ain Shams University 200
Early research (Phase 1)Active Not RecruitingNCT06436742What this trial is testingA Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)Who this might be right forCongenital Myasthenic SyndromeCMS argenx 15
Not applicableApproved For MarketingNCT00872950What this trial is testing3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)Who this might be right forLambert-Eaton Myasthenic SyndromeCongenital Myasthenic Syndrome Lahey Clinic
Not applicableLooking for participantsNCT07478172What this trial is testingEffects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseWho this might be right forNeuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more University of Missouri-Columbia 50
Not applicableStudy completedNCT05408702What this trial is testingExercise in Autoimmune Myasthenia Gravis and Myasthenic SyndromesWho this might be right forAutoimmune Myasthenia GravisLambert-Eaton Myasthenic SyndromeCongenital Myasthenic Syndrome+1 more Institut de Myologie, France 500
Not applicableEnrolling By InvitationNCT06605612What this trial is testingDevelopment and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular DisordersWho this might be right forInclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more LMU Klinikum 108
Large-scale testing (Phase 3)Not Yet RecruitingNCT07746089What this trial is testingAssess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)Who this might be right forCongenital Myasthenic SyndromeCMS argenx 105
Not applicableApproved For MarketingNCT02189720What this trial is testingExpanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic SyndromeWho this might be right forCongenital Myasthenic SyndromeLambert-Eaton Myasthenic SyndromeNystagmus, Acquired Catalyst Pharmaceuticals, Inc.
Early research (Phase 1)Not Yet RecruitingNCT07830563What this trial is testingEvaluating the Safety and Effects of AMP-101 in People With DOK7 Congenital Myasthenia SyndromeWho this might be right forCongenital Myasthenia Syndrome Amplo Biotechnology 4
Early research (Phase 1)Looking for participantsNCT07226726What this trial is testingPatients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome SolutionWho this might be right forCongenital Myasthenic Syndrome The Foundation for Orthopaedics and Regenerative Medicine 20
Early research (Phase 1)Study completedNCT01203592What this trial is testingEfficacy of Albuterol in the Treatment of Congenital Myasthenic SyndromesWho this might be right forCongenital Myasthenic Syndrome Mayo Clinic 21