Duchenne Muscular Dystrophy
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CLINICAL_TRIALS trial_result · 2024-02-14Clinical Trial Update: Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) [Phase 3]
Phase 3 trial update for viltolarsen in ambulant boys with Duchenne muscular dystrophy (DMD).
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CLINICAL_TRIALS trial_result · 2024-01-23Clinical Trial Update: TXA127 in Non-Ambulant Patients With DMD Cardiomyopathy [Phase 2]
Phase 2 clinical trial update for TXA127 in non-ambulant Duchenne muscular dystrophy–associated cardiomyopathy.
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CLINICAL_TRIALS trial_result · 2024-01-11Clinical Trial Update: Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval [Phase 1]
Phase 1 trial assessing placebo-corrected QT/QTc effects of ITF2357 (Givinostat) at therapeutic and supratherapeutic doses alongside moxiflo…
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CLINICAL_TRIALS trial_result · 2023-10-23Clinical Trial Update: Spironolactone Versus Prednisolone in DMD [Phase 1]
Phase 1 trial comparing spironolactone versus prednisolone in Duchenne muscular dystrophy was terminated; results posted.
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CLINICAL_TRIALS trial_result · 2023-10-12Clinical Trial Update: 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. [Phase 2]
A 48-week Open Label study evaluated the efficacy and safety of AMONDYS 45, EXONDYS 51, and VYONDYS 53 in Duchenne muscular dystrophy patien…
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CLINICAL_TRIALS trial_result · 2023-09-21Clinical Trial Update: Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Pilot dose-escalation trial evaluating canakinumab (ILARIS) in Duchenne muscular dystrophy; Phase 1/2, status not specified.
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) [Phase 1, Phase 2]
A randomized, double-blind, placebo-controlled Phase 1/2 study of delandistrogene moxeparvovec (SRP-9001) for Duchenne muscular dystrophy ha…
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CLINICAL_TRIALS trial_result · 2023-08-18Clinical Trial Update: A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) [Phase 2]
A Phase 2 trial evaluating safety, tolerability, and efficacy of eteplirsen in Duchenne Muscular Dystrophy participants who completed a prio…
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CLINICAL_TRIALS trial_result · 2023-02-02Clinical Trial Update: Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy [Phase 3]
Phase 3 trial evaluating givinostat in ambulant Duchenne muscular dystrophy completed with posted results.
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CLINICAL_TRIALS trial_result · 2022-12-20Clinical Trial Update: Tamoxifen in Duchenne Muscular Dystrophy [Phase 3]
Phase 3 clinical trial update for Tamoxifen in Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2022-11-18Clinical Trial Update: Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 extension study of NS-065/NCNP-01 in boys with Duchenne muscular dystrophy completed; results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-10-19Clinical Trial Update: Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) [Phase 4]
Phase 4 trial update assessing long-term use of viltolarsen in boys with Duchenne muscular dystrophy in clinical practice.
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CLINICAL_TRIALS trial_result · 2022-09-29Clinical Trial Update: Exploratory Study of NS-089/NCNP-02 in DMD [Phase 1, Phase 2]
An exploratory Phase 1/2 trial of NS-089/NCNP-02 in Duchenne muscular dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2022-09-28Clinical Trial Update: Bisoprolol in DMD Early Cardiomyopathy [Phase 2, Phase 3]
Clinical trial update for bisoprolol in Duchenne Muscular Dystrophy–related cardiomyopathy, currently in Phase 2/Phase 3.
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CLINICAL_TRIALS trial_result · 2022-09-23Clinical Trial Update: Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A Phase 1/2 clinical trial in boys with Duchenne muscular dystrophy has completed with results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-08-12Clinical Trial Update: Finding the Optimum Regimen for Duchenne Muscular Dystrophy [Phase 3]
Phase 3 trial update for Duchenne Muscular Dystrophy evaluating the optimum regimen; study completed with results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-07-13Clinical Trial Update: A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) [Phase 2]
A Phase 2 clinical trial evaluating vamorolone for Duchenne muscular dystrophy has completed with results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-06-21Clinical Trial Update: Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy [Phase 3]
Phase 3 study of edasalonexent in boys with Duchenne Muscular Dystrophy completed; results posted.
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CLINICAL_TRIALS trial_result · 2022-04-25Clinical Trial Update: Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy [Phase 1, Phase 2]
This update reports results from a Phase 1/2 gene transfer trial delivering rAAVrh74.MCK.GALGT2 for Duchenne muscular dystrophy; the study i…
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CLINICAL_TRIALS trial_result · 2022-04-05Clinical Trial Update: A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) [Phase 2]
Phase 2 study evaluating dystrophin level changes in participants with nonsense mutation Duchenne muscular dystrophy completed; results post…
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CLINICAL_TRIALS trial_result · 2022-04-05Clinical Trial Update: A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren [Phase 2]
Phase 2 study assessing dystrophin levels in nmDMD patients treated with ataluren has completed with results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-02-18Clinical Trial Update: Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD [NA]
Clinical trial update indicating termination for a study evaluating dynamic arm supports to promote activities of daily living in Duchenne M…
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CLINICAL_TRIALS trial_result · 2021-12-30Clinical Trial Update: Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) [Phase 2]
Phase 2 trial of pamrevlumab (FG-3019) in non-ambulatory Duchenne muscular dystrophy was terminated; results have been posted.
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CLINICAL_TRIALS trial_result · 2021-12-21Clinical Trial Update: Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents [Phase 1, Phase 2]
Clinical trial update for plus epicatechin in non-ambulatory adolescents with Duchenne muscular dystrophy, completed with posted results.
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CLINICAL_TRIALS trial_result · 2021-12-09Clinical Trial Update: Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping [Phase 2]
Phase 2 study of eteplirsen in young Duchenne Muscular Dystrophy patients amenable to exon 51 skipping has completed.