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Results for “Neuromuscular; Disorder, Hereditary”

1,859 trials

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Showing 20 of 1,859 results

Not applicableLooking for participantsNCT06610019
What this trial is testing

Cardiovascular Multimodality Imaging Study

Who this might be right for
Non-ischemic CardiomyopathyCardiomyopathiesHypertrophic Cardiomyopathy+7 more
Montefiore Medical Center 5,000
Not applicableStudy completedNCT04892628
What this trial is testing

Effects of Progressive Resistance Training Compared With Aerobic Training in Patients With Hemophilia

Who this might be right for
Quality of LifeMuscle Weakness
Dow University of Health Sciences 60
Testing effectiveness (Phase 2)Active Not RecruitingNCT04980872
What this trial is testing

The Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

Who this might be right for
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Merck Sharp & Dohme LLC 60
Testing effectiveness (Phase 2)Study completedNCT02579239
What this trial is testing

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)

Who this might be right for
Limb-Girdle Muscular DystrophiesFacioscapulohumeral Muscular Dystrophy
aTyr Pharma, Inc. 18
Testing effectiveness (Phase 2)Active Not RecruitingNCT04800874
What this trial is testing

Study of BBP-418 in Patients With LGMD2I

Who this might be right for
LGMD2I
ML Bio Solutions, Inc. 14
Not applicableStudy completedNCT00344890
What this trial is testing

Safety Study of Preservon-Treated Bone Implants for Cervical Fusion in the Treatment of Cervical Radiculopathy or Myelopathy

Who this might be right for
Cervical RadiculopathyMyelopathy
LifeNet Health 106
Large-scale testing (Phase 3)Active Not RecruitingNCT05680818
What this trial is testing

Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1

Who this might be right for
Autosomal Dominant Hypocalcemia (ADH)
Calcilytix Therapeutics, Inc., a BridgeBio company 67
Not applicableStudy completedNCT03440658
What this trial is testing

Deficits of Strength of the Rotator Muscles of the Shoulder in Children

Who this might be right for
Obstetrical Brachial Plexus Palsy
University Hospital, Montpellier 20
Large-scale testing (Phase 3)Ended earlyNCT04378075
What this trial is testing

Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Who this might be right for
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more
PTC Therapeutics 68
Testing effectiveness (Phase 2)Looking for participantsNCT06185764
What this trial is testing

VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Who this might be right for
Myotonic Dystrophy Type 1 (DM1)
Vertex Pharmaceuticals Incorporated 36
Not applicableStudy completedNCT03018184
What this trial is testing

Contractile Cross Sectional Areas and Muscle Strength in Patients With Congenital Myopathies

Who this might be right for
Inherited Muscle DiseasesCongenital MyopathyRYR1-myopathy
Rigshospitalet, Denmark 31
Testing effectiveness (Phase 2)Active Not RecruitingNCT03190915
What this trial is testing

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Who this might be right for
Juvenile Myelomonocytic LeukemiaNeurofibromatosis Type 1
National Cancer Institute (NCI) 10
Large-scale testing (Phase 3)Study completedNCT03837184
What this trial is testing

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Who this might be right for
Spinal Muscular Atrophy Type I
Novartis Gene Therapies 2
Not applicableStudy completedNCT04999735
What this trial is testing

Digital Biotyping of FSHD Patients and Controls

Who this might be right for
Muscular Dystrophy, Facioscapulohumeral
Centre for Human Drug Research, Netherlands 58
Not applicableLooking for participantsNCT06747884
What this trial is testing

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Who this might be right for
Congenital Myotonic DystrophyChildhood Myotonic DystrophyMyotonic Dystrophy
Virginia Commonwealth University 200
Testing effectiveness (Phase 2)Study completedNCT02531217
What this trial is testing

Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension

Who this might be right for
Facioscapulohumeral Muscular Dystrophy
aTyr Pharma, Inc. 9
Testing effectiveness (Phase 2)Active Not RecruitingNCT05291091
What this trial is testing

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

Who this might be right for
Becker Muscular Dystrophy
Edgewise Therapeutics, Inc. 244
Post-approval studies (Phase 4)Study completedNCT05073133
What this trial is testing

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)

Who this might be right for
Muscular Atrophy, Spinal
Novartis Pharmaceuticals 16
Not applicableEnrolling By InvitationNCT06606340
What this trial is testing

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 300
Not applicableEnded earlyNCT02930408
What this trial is testing

Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects

Who this might be right for
Steinert's Disease
Poitiers University Hospital 70
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