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type=\u0022text\/css\u0022 rel=\u0022stylesheet\u0022 href=\u0022\/\/d282kpwvnogo5m.cloudfront.net\/sites\/default\/files\/cdn\/css\/http\/css_Xg7z6oCTVgud_Q0huYz9x9iiD5H_2YPSJ5z2ZViSWdY.css\u0022 media=\u0022all\u0022 \/\u003E\n\u003Clink rel=\u0027stylesheet\u0027 type=\u0027text\/css\u0027 href=\u0027\/sites\/all\/modules\/contrib\/panels\/plugins\/layouts\/onecol\/onecol.css\u0027 \/\u003E\u003C\/head\u003E\u003Cbody\u003E\u003Cdiv class=\u0022panels-ajax-tab-panel panels-ajax-tab-panel-sageoa-tab-art\u0022\u003E\u003Cdiv class=\u0022panel-display panel-1col clearfix\u0022 \u003E\n  \u003Cdiv class=\u0022panel-panel panel-col\u0022\u003E\n    \u003Cdiv\u003E\u003Cdiv class=\u0022panel-pane pane-highwire-markup\u0022 \u003E\n  \n      \n  \n  \u003Cdiv class=\u0022pane-content\u0022\u003E\n    \u003Cdiv class=\u0022highwire-markup\u0022\u003E\u003Cdiv xmlns=\u0022http:\/\/www.w3.org\/1999\/xhtml\u0022 id=\u0022content-block-markup\u0022 xmlns:xhtml=\u0022http:\/\/www.w3.org\/1999\/xhtml\u0022\u003E\u003Cdiv class=\u0022article fulltext-view \u0022\u003E\u003Cspan class=\u0022highwire-journal-article-marker-start\u0022\u003E\u003C\/span\u003E\u003Cdiv class=\u0022section abstract\u0022 id=\u0022abstract-1\u0022\u003E\u003Ch2\u003ESummary\u003C\/h2\u003E\n            \u003Cp id=\u0022p-1\u0022\u003EThe randomized phase 3 DELOS trial was designed to evaluate idebenone, a synthetic quinone compound, in Duchenne muscular dystrophy. The data showed that idebenone was safe and well tolerated, and it significantly reduced loss of respiratory function in patients aged 10 to 18 years.\u003C\/p\u003E\n         \u003C\/div\u003E\u003Cul class=\u0022kwd-group\u0022\u003E\u003Cli class=\u0022kwd\u0022\u003Eidebenone\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003EDuchenne muscular dystrophy\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003Edystrophin deficiency\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003EDELOS\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003Erespiratory failure\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003Epeak expiratory flow\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003Eforced vital capacity\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003Eforced expiratory volume in 1 second\u003C\/li\u003E\u003Cli class=\u0022kwd\u0022\u003Eneurology clinical trials\u003C\/li\u003E\u003C\/ul\u003E\u003Cdiv class=\u0022section\u0022 id=\u0022sec-1\u0022\u003E\n         \n         \u003Cp id=\u0022p-2\u0022\u003EGunnar Buyse, MD, PhD, University of Leuven, Leuven, Belgium, presented data from a phase 3, double-blind, randomized, placebo-controlled study [DELOS; Buyse GM et al. \u003Cem\u003ELancet\u003C\/em\u003E. 2015] of the efficacy, safety, and tolerability of idebenone in patients aged 10 to 18 years with Duchenne muscular dystrophy (DMD). The results demonstrated that idebenone significantly reduced the decline of respiratory function in patients with DMD who were not receiving corticosteroids.\u003C\/p\u003E\n         \u003Cp id=\u0022p-3\u0022\u003EDMD is a devastating condition with no cure, and it is the most common type of muscular degenerative disease worldwide. Progressive respiratory failure is a major cause of morbidity in patients, and it is often the main cause of death in young adulthood.\u003C\/p\u003E\n         \u003Cp id=\u0022p-4\u0022\u003EAccording to Prof Buyse, in patients with DMD, dystrophin deficiency causes a calcium ion influx into muscle cells, resulting in mitochondrial dysfunction with reduced cellular energy production and increased formation of reactive oxygen species. Idebenone represents a novel treatment approach for DMD, he explained. It is a synthetic quinone compound that stimulates electron transfer and cellular energy production, and functions as an antioxidant, thereby counteracting some of the biochemical changes that occur in DMD.\u003C\/p\u003E\n         \u003Cp id=\u0022p-5\u0022\u003EBased on successful preclinical [Buyse GM et al. \u003Cem\u003EEur Heart J\u003C\/em\u003E. 2009] and phase 2 [Buyse GM et al. \u003Cem\u003EPediatr Pulmonol\u003C\/em\u003E. 2013; Buyse GM et al. \u003Cem\u003ENeuromusc Disord\u003C\/em\u003E. 2011] data, Prof Buyse and colleagues therefore conducted a multicenter, international, randomized controlled phase 3 trial (DELOS) to assess the efficacy and safety of idebenone in DMD.\u003C\/p\u003E\n         \u003Cp id=\u0022p-6\u0022\u003EThe study randomized and treated 64 patients aged between 10 and 18 years who were not currently receiving chronic corticosteroid treatment, of whom 92% were confined to wheelchairs.\u003C\/p\u003E\n         \u003Cp id=\u0022p-7\u0022\u003EPatients received either idebenone 900 mg\/day (n\u2005=\u200531) or placebo (n\u2005=\u200533) for 52 weeks. The primary end point was peak expiratory flow (a measure of respiratory strength) as a percentage of predicted normal (PEF%p). Secondary end points included forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV\u003Csub\u003E1\u003C\/sub\u003E).\u003C\/p\u003E\n         \u003Cp id=\u0022p-8\u0022\u003EAt 52 weeks, idebenone significantly reduced the decline in PEF%p compared with placebo in the modified intention-to-treat (ITT) population (\u20133.05 vs \u22129.01; \u003Cem\u003EP\u003C\/em\u003E\u2005=\u2005.04; \u003Ca id=\u0022xref-fig-1-1\u0022 class=\u0022xref-fig\u0022 href=\u0022#F1\u0022\u003EFigure 1\u003C\/a\u003E) and the ITT population (\u22122.57 vs \u22128.84; \u003Cem\u003EP\u003C\/em\u003E\u2005=\u2005.03), representing a reduction in loss of PEF%p of 66% and 71%, respectively.\u003C\/p\u003E\n         \u003Cdiv id=\u0022F1\u0022 class=\u0022fig pos-float  odd\u0022\u003E\u003Cdiv class=\u0022highwire-figure\u0022\u003E\u003Cdiv class=\u0022fig-inline-img-wrapper\u0022\u003E\u003Cdiv class=\u0022fig-inline-img\u0022\u003E\u003Ca href=\u0022http:\/\/d282kpwvnogo5m.cloudfront.net\/content\/spmdc\/15\/9\/6\/F1.large.jpg?width=800\u0026amp;height=600\u0026amp;carousel=1\u0022 title=\u0022Effect of Idebenone on Peak Expiratory Flow in mITT PopulationmITT, modified intention-to-treat; PEF%p, peak expiratory flow as a percentage of predicted normal.Reproduced with permission from G Buyse, MD, PhD.\u0022 class=\u0022fragment-images colorbox-load\u0022 rel=\u0022gallery-fragment-images-2075043417\u0022 data-figure-caption=\u0022Effect of Idebenone on Peak Expiratory Flow in mITT PopulationmITT, modified intention-to-treat; PEF%p, peak expiratory flow as a percentage of predicted normal.Reproduced with permission from G Buyse, MD, PhD.\u0022 data-icon-position=\u0022\u0022 data-hide-link-title=\u00220\u0022\u003E\u003Cimg class=\u0022fragment-image\u0022 alt=\u0022Figure 1.\u0022 src=\u0022http:\/\/d282kpwvnogo5m.cloudfront.net\/content\/spmdc\/15\/9\/6\/F1.medium.gif\u0022\/\u003E\u003C\/a\u003E\u003C\/div\u003E\u003C\/div\u003E\u003Cul class=\u0022highwire-figure-links inline\u0022\u003E\u003Cli class=\u00220 first\u0022\u003E\u003Ca href=\u0022http:\/\/d282kpwvnogo5m.cloudfront.net\/content\/spmdc\/15\/9\/6\/F1.large.jpg?download=true\u0022 class=\u0022highwire-figure-link highwire-figure-link-download\u0022 title=\u0022Download Figure 1.\u0022 data-icon-position=\u0022\u0022 data-hide-link-title=\u00220\u0022\u003EDownload figure\u003C\/a\u003E\u003C\/li\u003E\u003Cli class=\u00221\u0022\u003E\u003Ca href=\u0022http:\/\/d282kpwvnogo5m.cloudfront.net\/content\/spmdc\/15\/9\/6\/F1.large.jpg\u0022 class=\u0022highwire-figure-link highwire-figure-link-newtab\u0022 target=\u0022_blank\u0022 data-icon-position=\u0022\u0022 data-hide-link-title=\u00220\u0022\u003EOpen in new tab\u003C\/a\u003E\u003C\/li\u003E\u003Cli class=\u00222 last\u0022\u003E\u003Ca href=\u0022\/highwire\/powerpoint\/16579\u0022 class=\u0022highwire-figure-link highwire-figure-link-ppt\u0022 data-icon-position=\u0022\u0022 data-hide-link-title=\u00220\u0022\u003EDownload powerpoint\u003C\/a\u003E\u003C\/li\u003E\u003C\/ul\u003E\u003C\/div\u003E\u003Cdiv class=\u0022fig-caption\u0022 xmlns:xhtml=\u0022http:\/\/www.w3.org\/1999\/xhtml\u0022\u003E\u003Cspan class=\u0022fig-label\u0022\u003EFigure 1.\u003C\/span\u003E \n               \u003Cp id=\u0022p-9\u0022 class=\u0022first-child\u0022\u003EEffect of Idebenone on Peak Expiratory Flow in mITT Population\u003C\/p\u003E\n               \u003Cp id=\u0022p-10\u0022\u003EmITT, modified intention-to-treat; PEF%p, peak expiratory flow as a percentage of predicted normal.\u003C\/p\u003E\n               \u003Cp id=\u0022p-11\u0022\u003EReproduced with permission from G Buyse, MD, PhD.\u003C\/p\u003E\n            \u003Cdiv class=\u0022sb-div caption-clear\u0022\u003E\u003C\/div\u003E\u003C\/div\u003E\u003C\/div\u003E\n         \u003Cp id=\u0022p-12\u0022\u003EIdebenone also resulted in a 37% reduction in loss of FVC as a percentage of predicted normal, although not significantly (\u22125.67 vs \u22128.95; \u003Cem\u003EP\u003C\/em\u003E\u2005=\u2005.08), and a 78% reduction in loss of FEV\u003Csub\u003E1\u003C\/sub\u003E as a percentage of predicted normal (\u22122.40 vs \u221210.68; \u003Cem\u003EP\u003C\/em\u003E\u2005=\u2005.03) in the ITT population.\u003C\/p\u003E\n         \u003Cp id=\u0022p-13\u0022\u003EThis study represents the first successful phase 3 trial in DMD, and it demonstrates significant benefit of idebenone treatment in patients with this disease. Treatment with idebenone was also safe and well tolerated, concluded Prof Buyse.\u003C\/p\u003E\n      \u003C\/div\u003E\u003Cul class=\u0022copyright-statement\u0022\u003E\u003Cli class=\u0022fn\u0022 id=\u0022copyright-statement-1\u0022\u003E\u00a9 2015 SAGE Publications\u003C\/li\u003E\u003C\/ul\u003E\u003Cspan class=\u0022highwire-journal-article-marker-end\u0022\u003E\u003C\/span\u003E\u003C\/div\u003E\u003Cspan id=\u0022related-urls\u0022\u003E\u003C\/span\u003E\u003C\/div\u003E\u003Ca href=\u0022http:\/\/mdc.sagepub.com\/content\/15\/9\/6.abstract\u0022 class=\u0022hw-link hw-link-article-abstract\u0022 data-icon-position=\u0022\u0022 data-hide-link-title=\u00220\u0022\u003EView Summary\u003C\/a\u003E\u003C\/div\u003E  \u003C\/div\u003E\n\n  \n  \u003C\/div\u003E\n\u003C\/div\u003E\n  \u003C\/div\u003E\n\u003C\/div\u003E\n\u003C\/div\u003E\u003Cscript type=\u0022text\/javascript\u0022 src=\u0022http:\/\/mdc.sagepub.com\/sites\/all\/modules\/highwire\/highwire\/plugins\/highwire_markup_process\/js\/highwire_figures.js?nzlns1\u0022\u003E\u003C\/script\u003E\n\u003Cscript type=\u0022text\/javascript\u0022 src=\u0022http:\/\/mdc.sagepub.com\/sites\/all\/modules\/highwire\/highwire\/plugins\/highwire_markup_process\/js\/highwire_openurl.js?nzlns1\u0022\u003E\u003C\/script\u003E\n\u003C\/body\u003E\u003C\/html\u003E"}