Duchenne Muscular Dystrophy
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Sign up freeTop drugs studied for this disease
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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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FDA approval · 2022-01-18FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)
The FDA approved a supplemental application for Exondys 51 (eteplirsen) submitted by Sarepta Therapeutics, expanding its regulatory status.
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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.
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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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FDA label_change · 2021-06-29FDA Approved: EMFLAZA — Supplemental Application (PTC Therapeutics, Inc.)
FDA approved a supplemental application for Emflaza (deflazacort) by PTC Therapeutics, expanding the drug’s labeling. The update concerns Du…
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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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FDA approval · 2021-03-11FDA Approved: VILTEPSO — Supplemental Application (NS Pharma, Inc.)
The FDA approved a supplemental application for VILTEPSO (viltolarsen) from NS Pharma, Inc., resulting in updated labeling for Duchenne musc…