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Results for “Neuromuscular; Disorder, Hereditary”

1,984 trials

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Showing 20 of 1,984 results

Not applicableStudy completedNCT03588468
What this trial is testing

Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study

Who this might be right for
Amyloid NeuropathiesTransthyretin Amyloidosis
Assistance Publique Hopitaux De Marseille 60
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 applicableEnrolling By InvitationNCT06664866
What this trial is testing

AI Echocardiographic Screening of Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
Cedars-Sinai Medical Center 500
Not applicableNo Longer AvailableNCT04825613
What this trial is testing

"Individual Patient Expanded Access IND to Treat Primary Lateral Sclerosis"

Who this might be right for
Primary Lateral Sclerosis, Adult, 1
Hope Biosciences Research Foundation
Testing effectiveness (Phase 2)Study completedNCT00887562
What this trial is testing

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

Who this might be right for
MELAS Syndrome
Michio Hirano 27
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Large-scale testing (Phase 3)Ended earlyNCT02090959
What this trial is testing

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 219
Post-approval studies (Phase 4)Study completedNCT01070511
What this trial is testing

Tadalafil in Becker Muscular Dystrophy

Who this might be right for
Becker Muscular Dystrophy
Cedars-Sinai Medical Center 48
Not applicableActive Not RecruitingNCT05581511
What this trial is testing

Natural History Study of Cutaneous Neurofibromas in People With NF1

Who this might be right for
Neurofibromatosis Type 1Neurofibromatosis 1Neurofibromatosis (Nonmalignant)+1 more
Johns Hopkins University 500
Not applicableStudy completedNCT04661800
What this trial is testing

Study of Olfactory Disorders in Patients With Cardiac Amyloidosis

Who this might be right for
AmyloidosisCardiac AmyloidosisOlfactory Loss+4 more
Assistance Publique - Hôpitaux de Paris 51
Not applicableLooking for participantsNCT06880653
What this trial is testing

Dose Response of Exercise for Arthritis Management

Who this might be right for
ArthritisRheumatoid Arthritis (RA)Gout+3 more
University of South Carolina 285
Testing effectiveness (Phase 2)Study completedNCT00600275
What this trial is testing

A Phase I/II Study of BGT226 in Adult Patients With Advanced Solid Malignancies Including Patients With Advanced Breast Cancer

Who this might be right for
Solid TumorsBreast CancerCowden Syndrome
Novartis Pharmaceuticals 57
Not applicableLooking for participantsNCT07689331
What this trial is testing

Hungarian National Systemic Amyloidosis Registry

Who this might be right for
Systemic AmyloidosisATTR AmyloidosisAL Amyloidosis+1 more
Semmelweis University 300
Not applicableUnknownNCT02269865
What this trial is testing

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

Who this might be right for
Myotonic Dystrophy Type 1
Stanford University 40
Not applicableStudy completedNCT05330195
What this trial is testing

Bicycle Ergometer Training in Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Hacettepe University 23
Not applicableUnknownNCT03811301
What this trial is testing

[BrainConnexion] - Neurodevice Phase I Trial

Who this might be right for
TetraplegiaTetraplegia/TetraparesisSpinal Cord Injuries+4 more
National Neuroscience Institute 5
Not applicableLooking for participantsNCT00313677
What this trial is testing

Clinical Trial Readiness for the Dystroglycanopathies

Who this might be right for
Muscular Dystrophy
Katherine Mathews 190
Testing effectiveness (Phase 2)WithdrawnNCT03520751
What this trial is testing

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

Who this might be right for
Charcot-Marie-Tooth Neuropathy Type 1A
Nationwide Children's Hospital
Early research (Phase 1)Study completedNCT02710500
What this trial is testing

RAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

Who this might be right for
Dysferlinopathy
Sarepta Therapeutics, Inc. 2
Testing effectiveness (Phase 2)Looking for participantsNCT06138639
What this trial is testing

SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Who this might be right for
Duchenne Muscular Dystrophy
Solid Biosciences Inc. 60
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