Duchenne Muscular Dystrophy
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CLINICAL_TRIALS withdrawal · 2014-03-24Clinical Trial Update: Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol [Phase 3]
The Phase 3 clinical trial of Drisapersen for Duchenne muscular dystrophy has been withdrawn by sponsor GlaxoSmithKline.
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CLINICAL_TRIALS trial_result · 2014-03-10Clinical Trial Update: KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy [Phase 2]
An open-label Phase 2 pilot study evaluating Oxatomide in steroid-naive Duchenne muscular dystrophy. The trial status is completed.
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CLINICAL_TRIALS trial_result · 2014-02-03Clinical Trial Update: Safety Study of Flavocoxid in Duchenne Muscular Dystrophy [Phase 1]
A Phase 1 safety study evaluating Flavocoxid in Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2014-01-28Clinical Trial Update: PDE Inhibitors in DMD Study (Acute Dosing Study) [Phase 1]
Phase 1 acute dosing study of PDE inhibitors in Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2013-12-09Clinical Trial Update: DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls [NA]
Clinical trial update noting the DART Electrical Impedance Myography trial in Duchenne Muscular Dystrophy (and healthy controls) completed w…
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CLINICAL_TRIALS trial_result · 2013-11-13Clinical Trial Update: Optimum Insufflation Capacity in NMD [NA]
This ClinicalTrials.gov update for NCT01981915 indicates completion of a study on optimum insufflation capacity in neuromuscular diseases, i…
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CLINICAL_TRIALS trial_result · 2013-10-11Clinical Trial Update: CoQ10 and Prednisone in Non-Ambulatory DMD [Phase 3]
Phase 3 trial of CoQ10 in combination with Prednisone in non-ambulatory Duchenne muscular dystrophy was terminated; results were posted in 2…
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CLINICAL_TRIALS trial_result · 2013-02-05Clinical Trial Update: Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy [Phase 1]
Phase 1 safety study of a mini-dystrophin gene therapy for Duchenne Muscular Dystrophy has been completed.
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CLINICAL_TRIALS trial_result · 2013-02-01Clinical Trial Update: Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy [Phase 2]
The Phase 2 extension study of ACE-031 for Duchenne muscular dystrophy has been terminated.
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CLINICAL_TRIALS trial_result · 2013-01-11Clinical Trial Update: Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
Completed comparative study in Duchenne muscular dystrophy evaluating handheld myometry versus CINRG CQMS as clinical endpoints.
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CLINICAL_TRIALS trial_result · 2013-01-11Clinical Trial Update: Cardiac Outcome Measures in Children With Muscular Dystrophy
A completed clinical trial (NCT01066455) evaluating cardiac outcome measures in children with Duchenne muscular dystrophy, Becker muscular d…
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CLINICAL_TRIALS trial_result · 2013-01-11Clinical Trial Update: Cardiac Magnetic Resonance in Children With Muscular Dystrophy
A completed clinical trial evaluated cardiac magnetic resonance imaging in children with muscular dystrophy to study cardiac involvement.
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CLINICAL_TRIALS trial_result · 2012-11-30Clinical Trial Update: Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy [Phase 1, Phase 2]
This document provides an update on a Phase 1/2 clinical trial evaluating the safety and efficacy of umbilical cord mesenchymal stem cell th…
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CLINICAL_TRIALS trial_result · 2012-10-02Clinical Trial Update: CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers [Phase 2]
Phase 2 trial update confirms completion of the CRD007 study evaluating treatment for Duchenne muscular dystrophy and Becker muscular dystro…
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CLINICAL_TRIALS trial_result · 2012-07-03Clinical Trial Update: Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids [Phase 1]
A Phase 1 clinical trial evaluating the safety, tolerability, and effects of L-Arginine in boys with dystrophinopathy on corticosteroids, in…
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CLINICAL_TRIALS trial_result · 2012-03-23Clinical Trial Update: Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons [Phase 1]
Six-month Phase 1 study evaluating gentamicin as a stop-codon read-through therapy for Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2011-10-27Clinical Trial Update: High-dose Prednisone in Duchenne Muscular Dystrophy [Phase 3]
Phase 3 trial update indicates completion of a high-dose prednisone study in Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS trial_result · 2011-10-27Clinical Trial Update: Pentoxifylline in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
A Phase 1/2 trial of pentoxifylline in Duchenne muscular dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2011-10-27Clinical Trial Update: Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy [Phase 2, Phase 3]
This update reports completion of a phase 2/3 trial evaluating creatine and L-glutamine in steroid-naive Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2011-10-27Clinical Trial Update: Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Completed Phase 1/2 clinical trial evaluating daily pentoxifylline as a rescue treatment for Duchenne muscular dystrophy. The study was spon…
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CLINICAL_TRIALS trial_result · 2011-07-29Clinical Trial Update: Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 trial evaluating the efficacy and tolerability of idebenone in boys with Duchenne Muscular Dystrophy-related cardiac dysfunction; th…
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CLINICAL_TRIALS trial_result · 2011-06-01Clinical Trial Update: Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) [Phase 2]
Long-term safety, tolerability, and efficacy data from the DELPHI extension of idebenone in Duchenne muscular dystrophy (Phase 2).
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CLINICAL_TRIALS trial_result · 2010-11-16Clinical Trial Update: An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy [Phase 2]
This report notes the completion of an open-label Phase 2 pilot trial evaluating Coenzyme Q10 in steroid-treated Duchenne muscular dystrophy…
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CLINICAL_TRIALS trial_result · 2009-01-14Clinical Trial Update: Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy [Phase 2]
A Phase 2 clinical trial evaluating the safety and efficacy of PTC124 (ataluren) in Duchenne muscular dystrophy has completed.
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: Gentamicin Treatment of Muscular Dystrophy [Phase 1]
This clinical trial update notes that a Phase 1 study of gentamicin for Becker Muscular Dystrophy and Duchenne Muscular Dystrophy has comple…