Found 12 matching trials
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) PHASE2 The purpose of this extension study is to evaluate the ongoing safety and t...
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Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy PHASE2 This is an open-label study to assess the safety, tolerability, efficacy an...
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Neuromuscular Research Center of Arizona
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Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients PHASE2 This study is designed to assess the efficacy, safety, tolerability, and ph...
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Nationwide Children's Hospital
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A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) PHASE3 Part 1 (dose escalation) will evaluate the safety and tolerability of 2 dos...
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Active Not Recruiting
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University of Alabama at Birmingham
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Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy PHASE1 Duchenne muscular dystrophy (DMD), a fatal muscle degenerative disorder, ar...
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Dubowitz Neuromuscular Centre, Hammersmith Hospital and Clinical Trails Unit, St Mary's Hospital
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A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. PHASE2 This is an 48-week open-label study to determine the efficacy and safety of...
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Nationwide Children's Hospital
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Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy PHASE2 The primary objective of this study is to assess the ongoing efficacy, safe...
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Miller Children's Hospital
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Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy PHASE2 The primary objective of this study is to explore safety and tolerability o...
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Ronald Reagan UCLA Medical Center
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Study of Eteplirsen in DMD Patients PHASE3 The main objective of this study is to provide evidence of efficacy of etep...
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Neuromuscular Research Center
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Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping PHASE2 This is a multicenter, open-label, dose-escalation study to evaluate the sa...
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Universitair ziekenhuis Gent
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