Duchenne Muscular Dystrophy
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CLINICAL_TRIALS trial_result · 2021-12-03Clinical Trial Update: Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) [Phase 3]
Phase III study of idebenone in Duchenne Muscular Dystrophy was terminated.
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CLINICAL_TRIALS trial_result · 2021-12-03Clinical Trial Update: A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids [Phase 3]
Phase III double-blind trial of idebenone in Duchenne Muscular Dystrophy patients on glucocorticoids; study terminated.
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CLINICAL_TRIALS trial_result · 2021-10-04Clinical Trial Update: The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD [Phase 3]
Phase 3 trial evaluating metoprolol as add-on therapy to standard of care for preventing cardiomyopathy in Duchenne muscular dystrophy patie…
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CLINICAL_TRIALS trial_result · 2021-07-29Clinical Trial Update: Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy [Phase 2, Phase 3]
A Phase 2/3 clinical trial of Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2021-07-12Clinical Trial Update: Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 safety and dose-finding study of NS-065/NCNP-01 in boys with Duchenne muscular dystrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-06-04Clinical Trial Update: A Study of TAS-205 for Duchenne Muscular Dystrophy [Phase 1]
Phase 1 study update for TAS-205 in Duchenne Muscular Dystrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-05-20Clinical Trial Update: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy [Phase 2, Phase 3]
Clinical trial update for WVE-210201 (Suvodirsen) in Duchenne muscular dystrophy shows termination after Phase 2/3 with results posted.
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CLINICAL_TRIALS trial_result · 2021-05-20Clinical Trial Update: Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Long-term extension study assessing vamorolone in boys with Duchenne muscular dystrophy has completed Phase 2 trial and results were posted …
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CLINICAL_TRIALS trial_result · 2021-05-17Clinical Trial Update: Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients [Phase 1]
Phase 1 dose-titration and open-label extension study of SRP-4045 in advanced Duchenne Muscular Dystrophy completed; results posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-02-21Clinical Trial Update: A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial of MNK-1411 in Duchenne Muscular Dystrophy was terminated; results posted.
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CLINICAL_TRIALS trial_result · 2021-01-29Clinical Trial Update: Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients [Phase 1, Phase 2]
Phase 1/2 clinical trial investigating transplantation of myoblasts in Duchenne Muscular Dystrophy patients.
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CLINICAL_TRIALS trial_result · 2021-01-20Clinical Trial Update: Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A safety and efficacy study of IGF-1 in Duchenne Muscular Dystrophy completed phases 1 and 2.
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CLINICAL_TRIALS trial_result · 2020-12-21Clinical Trial Update: Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy [Phase 2, Phase 3]
Phase 2/3 trial evaluating RO7239361 in ambulatory boys with Duchenne muscular dystrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-11-25Clinical Trial Update: Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada [Phase 3]
Phase 3 trial update for ataluren in previously treated nmDBMD participants with nonsense mutation Duchenne/Becker muscular dystrophy; study…
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CLINICAL_TRIALS trial_result · 2020-11-23Clinical Trial Update: An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy [Phase 3]
Open-label extension study update for edasalonexent in boys with Duchenne muscular dystrophy; Phase 3 trial terminated.
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CLINICAL_TRIALS trial_result · 2020-10-29Clinical Trial Update: Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2a extension study of ataluren (PTC124) in Duchenne Muscular Dystrophy was terminated; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Flu Vaccine Study in Neuromuscular Patients 2011 [Phase 4]
A Phase 4 clinical trial update on a flu vaccine study in pediatric neuromuscular disease patients has completed, with results posted in 202…
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Phase I/II Study of SRP-4053 in DMD Patients [Phase 1, Phase 2]
Phase I/II study update for SRP-4053 in Duchenne Muscular Dystrophy completed with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-08-28Clinical Trial Update: Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial of ataluren in young boys with Duchenne muscular dystrophy completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-08-11Clinical Trial Update: An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy [Phase 3]
Phase 3 extension study for ataluren (PTC124) in nonsense mutation dystrophinopathy was terminated; results were posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-08-04Clinical Trial Update: Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) [Phase 3]
Phase 3 trial of ataluren in nonsense mutation Duchenne muscular dystrophy completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-07-29Clinical Trial Update: Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) [Phase 2]
Phase 2 trial update for Ataluren (PTC124) in nonambulatory Duchenne/Becker muscular dystrophy due to nonsense mutations; study terminated w…
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CLINICAL_TRIALS trial_result · 2020-07-22Clinical Trial Update: Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy [Phase 2]
Phase 2 safety study of eteplirsen in early-stage Duchenne Muscular Dystrophy completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-07-15Clinical Trial Update: Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) [Phase 2]
Phase 2B extension study of ataluren in Duchenne/Becker muscular dystrophy was terminated; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-07-01Clinical Trial Update: Study of Eteplirsen in DMD Patients [Phase 3]
Phase 3 trial evaluating eteplirsen in Duchenne Muscular Dystrophy has completed with results posted.