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Results for “Dystrophinopathy”

10 trials

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Showing 10 of 10 results

Testing effectiveness (Phase 2)Study completedNCT02819557
What this trial is testing

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 14
Large-scale testing (Phase 3)Study completedNCT01247207
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
Large-scale testing (Phase 3)Study completedNCT01826487
What this trial is testing

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 230
Large-scale testing (Phase 3)Study completedNCT03179631
What this trial is testing

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 360
Large-scale testing (Phase 3)Study completedNCT01557400
What this trial is testing

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular DystrophyDystrophinopathy
PTC Therapeutics 94
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
Not applicableStudy completedNCT02411370
What this trial is testing

Correlation Between Respiratory Impairment and Phonemes Alteration in Dystrophinopathy Patients With Respiratory Failure

Who this might be right for
During the Medical Appointment, the Speech Therapist Will Perform Phonetic Tests
Centre d'Investigation Clinique et Technologique 805 30
Early research (Phase 1)Study completedNCT01388764
What this trial is testing

Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids

Who this might be right for
DystrophinopathyDuchenne Muscular DystrophyBecker's Muscular Dystrophy
Massachusetts General Hospital 7
Not applicableLooking for participantsNCT07515235
What this trial is testing

DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies

Who this might be right for
Duchenne Muscular Dystrophy (DMD)Becker Muscular DystrophyCardiomyopathy
Aristotle University Of Thessaloniki 65
Not applicableNot Yet RecruitingNCT07402122
What this trial is testing

Registry for Duchenne and Becker Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy (DMD)Becker Muscular DystrophyDystrophinopathy Symptomatic Female Carrier
Dr. Andreas Ziegler 1,500