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Results for “Congenital Myasthenic Syndrome”

23 trials

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Showing 20 of 23 results

Early research (Phase 1)Study completedNCT07105371
What this trial is testing

Patients With ALS and Other Motor Disorders Will be Treated With Mesenchymal Cell Exosome Solution

Who this might be right for
Motor Disorders
The Foundation for Orthopaedics and Regenerative Medicine 18
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 applicableNo Longer AvailableNCT03062631
What this trial is testing

Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia

Who this might be right for
Congenital Myasthenic Syndrome
Ricardo Maselli
Early research (Phase 1)Looking for participantsNCT06436742
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 16
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 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
Not applicableApproved For MarketingNCT02189720
What this trial is testing

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

Who this might be right for
Congenital Myasthenic SyndromeLambert-Eaton Myasthenic SyndromeNystagmus, Acquired
Catalyst Pharmaceuticals, Inc.
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 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 applicableStudy completedNCT01474980
What this trial is testing

Pregnancy Outcomes in Congenital Myasthenie Syndrome

Who this might be right for
Congenital Myasthenic Syndrome
Institut de Myologie, France 25
Not applicableStudy completedNCT05408702
What this trial is testing

Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes

Who this might be right for
Autoimmune Myasthenia GravisLambert-Eaton Myasthenic SyndromeCongenital Myasthenic Syndrome+1 more
Institut de Myologie, France 500
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
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 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 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
Large-scale testing (Phase 3)Study completedNCT02562066
What this trial is testing

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

Who this might be right for
Myasthenic Syndromes, Congenital
Catalyst Pharmaceuticals, Inc. 20
Not applicableNo Longer AvailableNCT01378546
What this trial is testing

Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP

Who this might be right for
Lambert Eaton Myasthenic Syndrome (LEMS)
Louis H. Weimer, MD
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
Early research (Phase 1)Looking for participantsNCT07226726
What this trial is testing

Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution

Who this might be right for
Congenital Myasthenic Syndrome
The Foundation for Orthopaedics and Regenerative Medicine 20
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