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Results for “Congenital Myasthenic Syndrome”

24 trials

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Showing 20 of 24 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 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 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 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
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 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
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
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 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 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
Early research (Phase 1)Study completedNCT01203592
What this trial is testing

Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes

Who this might be right for
Congenital Myasthenic Syndrome
Mayo Clinic 21
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 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 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 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 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
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 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
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