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Additiv zur bisherigen\n                    multidisziplin\u00e4ren supportiven pulmonalen, gastroenterologischen,\n                    orthop\u00e4dischen, neurop\u00e4diatrischen und neuromuskul\u00e4ren\n                    Behandlung wurden in den letzten Jahren 3 bahnbrechende\n                    erkrankungsmodifizierende Therapien der 5q-assoziierten spinalen Muskelatrophie\n                    (SMA) zugelassen, die Ph\u00e4notypen und Therapielandschaft entscheidend\n                    ver\u00e4ndert und damit neue Standards f\u00fcr die Beeinflussung von\n                    Neurodegeneration erm\u00f6glicht haben: Nusinersen\/Spinraza als\n                    Antisense-Oligonukleotidtherapie, Onasemnogene abeparvovec\/Zolgensma als\n                    eine AAV9-basierte Genersatztherapie, und Risdiplam\/Evrysdi als ein\n                    \u201esmall Molecule Modifier\u201c des pre-mRNA Splicings.<\/jats:p>","DOI":"10.1055\/a-1801-3785","type":"journal-article","created":{"date-parts":[[2022,9,14]],"date-time":"2022-09-14T22:46:37Z","timestamp":1663195597000},"page":"421-430","source":"Crossref","is-referenced-by-count":3,"title":["Spinale Muskelatrophie im Erwachsenenalter"],"prefix":"10.1055","volume":"90","author":[{"given":"Maggie C.","family":"Walter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Hiebeler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2022,9,14]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/j.nmd.2017.11.005","article-title":"Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for\n                    diagnosis, rehabilitation, orthopedic and nutritional care","volume":"28","author":"E Mercuri","year":"2018","journal-title":"Neuromuscul Disord"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27519,","article-title":"WeissMD. 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