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Results for “Dystrophinopathy”

9 trials

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Showing 9 of 9 results

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
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
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
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
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 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
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
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