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Results for “Congenital Myopathy”

986 trials

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Showing 20 of 986 results

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)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
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
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
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
Large-scale testing (Phase 3)Study completedNCT00004646
What this trial is testing

Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
National Center for Research Resources (NCRR) 20
Large-scale testing (Phase 3)Ended earlyNCT00308113
What this trial is testing

CoQ10 and Prednisone in Non-Ambulatory DMD

Who this might be right for
Duchenne Muscular Dystrophy
Cooperative International Neuromuscular Research Group 3
Testing effectiveness (Phase 2)Ended earlyNCT02927080
What this trial is testing

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

Who this might be right for
Facioscapulohumeral Muscular Dystrophy
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA 95
Not applicableNot Yet RecruitingNCT06874166
What this trial is testing

Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders

Who this might be right for
Duchenne / Becker Muscular DystrophyOsteogenesis Imperfecta (OI)Social Cognition
IRCCS Eugenio Medea 45
Large-scale testing (Phase 3)Enrolling By InvitationNCT01247207
What this trial is testing

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 270
Testing effectiveness (Phase 2)Ended earlyNCT01847573
What this trial is testing

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Processa Pharmaceuticals 17
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