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Grundlage der Therapie von Patienten mit DMD in jedem Krankheitsstadium sind supportive und symptomatische Ma\u00dfnahmen, die in der Regel auch nach dem Verlust der Gehf\u00e4higkeit intensiv weitergef\u00fchrt werden sollten. Zus\u00e4tzlich stehen den Patienten medikament\u00f6se Therapien mit dem Ziel der Modifikation des Krankheitsverlaufes zur Verf\u00fcgung. Glukokortikoide bilden den St\u00fctzpfeiler der medikament\u00f6sen Therapie auch \u00fcber den Verlust der Gehf\u00e4higkeit hinaus, dann meist in reduzierter Dosis. F\u00fcr Patienten mit DMD aufgrund einer Nonsense-Mutation (nmDMD), ca.\u00a013\u202f% aller DMD-Patienten, steht Ataluren als potenziell dystrophinwiederherstellende, krankheitsmodifizierende Therapie zur Verf\u00fcgung; klinische Daten aus dem STRIDE-Register zeigen eine verz\u00f6gerte Krankheitsprogression auch nach Verlust der Gehf\u00e4higkeit. Zum Exon-Skipping liegen f\u00fcr erwachsene Patienten derzeit noch keine belastbaren Daten vor. Das Antioxidans Idebenon kommt bei nichtgehf\u00e4higen, jugendlichen Patienten ohne therapeutische Alternative, die nicht mit Glukokortikoiden behandelt werden k\u00f6nnen, infrage. Ataluren eignet sich zur kombinierten Behandlung mit Glukokortikoiden, eine Kombination von Idebenon und Glukokortikoiden wird derzeit in einer klinischen Studie \u00fcberpr\u00fcft. Eine Add-on-Therapie mit Idebenon zus\u00e4tzlich zu Ataluren ist bei nichtgehf\u00e4higen nmDMD-Patienten zu erw\u00e4gen. Bedingt durch die Tatsache, dass sich einige der diskutierten Therapieoptionen noch in der Phase der klinischen Pr\u00fcfung befinden oder noch keine oder nur begrenzte Daten f\u00fcr \u00e4ltere Patienten mit DMD vorliegen, handelt es sich um Expertenempfehlungen entsprechend der Evidenzklasse\u00a0IV.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s00115-020-01019-3","type":"journal-article","created":{"date-parts":[[2020,11,19]],"date-time":"2020-11-19T22:22:09Z","timestamp":1605824529000},"page":"359-366","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Expertenempfehlung: Therapie nichtgehf\u00e4higer Patienten mit Muskeldystrophie Duchenne","Expert recommendation: treatment of nonambulatory patients with Duchenne muscular dystrophy"],"prefix":"10.1007","volume":"92","author":[{"given":"Guenther","family":"Bernert","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Hahn","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cornelia","family":"K\u00f6hler","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sascha","family":"Meyer","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ulrike","family":"Schara","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kurt","family":"Schlachter","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Regina","family":"Trollmann","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maggie C.","family":"Walter","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2020,11,19]]},"reference":[{"key":"1019_CR1","doi-asserted-by":"publisher","first-page":"e15858","DOI":"10.1097\/MD.0000000000015858","volume":"98","author":"LN Alfano","year":"2019","unstructured":"Alfano\u00a0LN, Charleston\u00a0JS, Connolly\u00a0AM et al (2019) Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. 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