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Results for “Duchenne Muscular Dystrophin (DMD)”

38 trials

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Showing 20 of 38 results

Testing effectiveness (Phase 2)Study completedNCT03796637
What this trial is testing

Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 6
Not applicableEnrolling By InvitationNCT05715957
What this trial is testing

Follow-up Study on Female Carriers With DMD Gene Variants

Who this might be right for
Muscular DystrophyDuchenne Muscular DystrophyBecker Muscular Dystrophy
Rigshospitalet, Denmark 103
Not applicableUnknownNCT02020954
What this trial is testing

Prospective Becker-Heart-Study

Who this might be right for
Dilated CardiomyopathyLef Ventricular DysfunctionHeart Failure
Karim WAHBI 100
Not applicableAvailableNCT07250737
What this trial is testing

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

Who this might be right for
Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal Diseases+6 more
Avidity Biosciences, Inc.
Testing effectiveness (Phase 2)WithdrawnNCT06833931
What this trial is testing

PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
PepGen Inc
Testing effectiveness (Phase 2)Ended earlyNCT05429372
What this trial is testing

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Pfizer 10
Testing effectiveness (Phase 2)Study completedNCT02286947
What this trial is testing

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Sarepta Therapeutics, Inc. 24
Testing effectiveness (Phase 2)Looking for participantsNCT07287189
What this trial is testing

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Who this might be right for
Duchenne Muscular DystrophyDuchenneDMD+2 more
Satellos Bioscience, Inc. 51
Large-scale testing (Phase 3)Study completedNCT03703882
What this trial is testing

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Catabasis Pharmaceuticals 131
Early research (Phase 1)Study completedNCT00005574
What this trial is testing

Gentamicin Treatment of Muscular Dystrophy

Who this might be right for
Becker Muscular DystrophyDuchenne Muscular Dystrophy
National Institute of Neurological Disorders and Stroke (NINDS) 4
Testing effectiveness (Phase 2)Active Not RecruitingNCT06128564
What this trial is testing

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
Hoffmann-La Roche 13
Testing effectiveness (Phase 2)Study completedNCT01540409
What this trial is testing

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics, Inc. 12
Early research (Phase 1)Looking for participantsNCT04626674
What this trial is testing

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 83
Testing effectiveness (Phase 2)UnknownNCT05753462
What this trial is testing

Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Sqy Therapeutics 12
Testing effectiveness (Phase 2)Looking for participantsNCT07673809
What this trial is testing

Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
AO GENERIUM 32
Not applicableStudy completedNCT03879304
What this trial is testing

Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker.

Who this might be right for
Muscular Dystrophy, Duchenne and Becker Types
University of Malaga 12
Very early researchActive Not RecruitingNCT06594094
What this trial is testing

An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)

Who this might be right for
Duchenne Muscular Dystrophin (DMD)
HuidaGene Therapeutics Co., Ltd. 4
Testing effectiveness (Phase 2)Ended earlyNCT02958202
What this trial is testing

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
BioMarin Pharmaceutical 7
Not applicableWithdrawnNCT05558813
What this trial is testing

Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)

Who this might be right for
Duchenne Muscular Dystrophy
Assistance Publique - Hôpitaux de Paris
Testing effectiveness (Phase 2)Study completedNCT03375164
What this trial is testing

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. 4
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