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Duchenne Muscular Dystrophy | "Dystrophinopathy"
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Duchenne Muscular Dystrophy | "Dystrophinopathy" (8 records)
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1 | NCT02109692 | Recruiting | Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies |
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Interventional | Not Applicable |
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Other |
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186 | All | 18 Months to 80 Years (Child, Adult, Senior) | NCT02109692 | 9184 | biodystromirs | May 2014 | November 2017 | May 2018 | April 10, 2014 | January 9, 2015 |
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2 | NCT01557400 | Active, not recruiting | Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada |
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Interventional | Phase 3 |
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Industry |
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95 | Male | Child, Adult, Senior | NCT01557400 | PTC124-GD-019-DMD | May 2012 | March 2018 | March 2018 | March 19, 2012 | January 18, 2018 |
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3 | NCT01388764 | Completed | Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids |
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Interventional | Phase 1 |
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Other |
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7 | Male | 7 Years to 11 Years (Child) | NCT01388764 | 2011D001591 | January 2012 | May 2012 | May 2012 | July 7, 2011 | July 3, 2012 |
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4 | NCT02819557 | Active, not recruiting | Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy |
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Interventional | Phase 2 |
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Industry |
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14 | Male | 2 Years to 5 Years (Child) | NCT02819557 | PTC124-GD-030-DMD | June 2016 | October 2017 | October 2017 | June 30, 2016 | June 1, 2017 |
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5 | NCT01826487 | Completed | Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy |
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Interventional | Phase 3 |
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Industry |
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230 | Male | 7 Years to 16 Years (Child) | NCT01826487 | PTC124-GD-020-DMD | ACT DMD | March 2013 | August 2015 | August 2015 | April 8, 2013 | December 13, 2017 |
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6 | NCT01247207 | Enrolling by invitation | Study of Ataluren for Previously Treated Patients With nmDBMD in the US |
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Interventional | Phase 3 |
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Industry |
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110 | Male | Child, Adult, Senior | NCT01247207 | PTC124-GD-016 DMD | November 2010 | December 2018 | December 2018 | November 24, 2010 | April 26, 2018 |
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7 | NCT02090959 | Active, not recruiting | Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy |
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Interventional | Phase 3 |
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Industry |
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219 | Male | 7 Years to 18 Years (Child, Adult) | NCT02090959 | PTC124-GD-020e-DMD | March 2014 | June 2018 | June 2018 | March 19, 2014 | January 18, 2018 |
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8 | NCT03179631 | Recruiting | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy |
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Interventional | Phase 3 |
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Industry |
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250 | Male | 5 Years and older (Child, Adult, Senior) | NCT03179631 | PTC124-GD-041-DMD | July 6, 2017 | March 2020 | December 2021 | June 7, 2017 | March 30, 2018 |
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† Study has passed its completion date and status has not been verified in more than two years.