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Results for “Congenital Myasthenia Syndrome”

34 trials

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Showing 20 of 34 results

Early research (Phase 1)Active Not RecruitingNCT06436742
What this trial is testing

A 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 for
Congenital Myasthenic SyndromeCMS
argenx 15
Not applicableApproved For MarketingNCT00872950
What this trial is testing

3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)

Who this might be right for
Lambert-Eaton Myasthenic SyndromeCongenital Myasthenic Syndrome
Lahey Clinic
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The 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 for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
Not applicableStudy completedNCT05945784
What this trial is testing

Exploring Accessible Beauty for Individuals With Upper Extremity Deficits

Who this might be right for
Stroke, IschemicStroke HemorrhagicStroke (CVA) or TIA+11 more
Casa Colina Hospital and Centers for Healthcare 57
Not applicableStudy completedNCT05890833
What this trial is testing

The Risk of Falls Index for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyFacioscapulohumeral Muscular Dystrophies+3 more
LMU Klinikum 30
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Very early researchLooking for participantsNCT04561557
What this trial is testing

Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System

Who this might be right for
Autoimmune DiseasesAutoimmune Diseases of the Nervous SystemNeuromyelitis Optica Spectrum Disorder+7 more
Tongji Hospital 36
Testing effectiveness (Phase 2)UnknownNCT00541216
What this trial is testing

Ephedrine for the Treatment of Congenital Myasthenia

Who this might be right for
Myasthenic Syndromes, Congenital
Hadassah Medical Organization 15
Not applicableLooking for participantsNCT07136844
What this trial is testing

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Who this might be right for
Neuromuscular DiseasesObesity (Disorder)Myotonic Dystrophy 1+9 more
Centre Hospitalier Universitaire de Liege 300
Not applicableLooking for participantsNCT07478172
What this trial is testing

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Who this might be right for
Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more
University of Missouri-Columbia 50
Not applicableUnknownNCT05635266
What this trial is testing

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Who this might be right for
Age-Related Macular DegenerationAllergiesAlpha-Gal Syndrome+44 more
Sanguine Biosciences 20,000
Not applicableUnknownNCT02124616
What this trial is testing

National Registry for Egyptian Pediatric Neuromuscular Diseases

Who this might be right for
Spinal Muscular AtrophyMuscular DystrophyMuscle Diseases+2 more
Ain Shams University 200
Large-scale testing (Phase 3)Not Yet RecruitingNCT07746089
What this trial is testing

Assess 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 for
Congenital Myasthenic SyndromeCMS
argenx 105
Not applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableLooking for participantsNCT01403402
What this trial is testing

Congenital Muscle Disease Study of Patient and Family Reported Medical Information

Who this might be right for
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) DeficiencyAlpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations)+49 more
Cure CMD
Not applicableNo Longer AvailableNCT02012933
What this trial is testing

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

Who this might be right for
Lambert-Eaton Myasthenic Syndrome (LEMS)Congenital Myasthenia (CM)
Oregon Health and Science University
Not applicableLooking for participantsNCT06630650
What this trial is testing

A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes

Who this might be right for
Myasthenic Syndromes, Congenital
National Institute of Neurological Disorders and Stroke (NINDS) 75
Not applicableNo Longer AvailableNCT01765140
What this trial is testing

Treatment Use of 3,4-Diaminopyridine

Who this might be right for
Myasthenic Syndromes, Congenital
Vern C. Juel, M.D.
Not applicableLooking for participantsNCT06078553
What this trial is testing

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

Who this might be right for
Congenital Myasthenic Syndrome
argenx 100
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