{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T21:50:19Z","timestamp":1778536219971,"version":"3.51.4"},"reference-count":0,"publisher":"Georg Thieme Verlag KG","issue":"12","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Fortschr Neurol Psychiatr"],"published-print":{"date-parts":[[2019,12]]},"abstract":"<jats:title>Zusammenfassung<\/jats:title><jats:p>Seit 2017 steht mit Nusinersen die erste kausale Therapie der 5q-assoziierten spinalen Muskelatrophie (SMA) zur Verf\u00fcgung. Erfahrungen aus der Praxis an neuromuskul\u00e4ren Zentren in Deutschland zeigen zunehmend den therapeutischen Nutzen von Nusinersen auch bei erwachsenen SMA-Patienten beiderlei Geschlechts: Vielfach gelingt eine Stabilisierung bzw. eine relevante Verbesserung motorischer Funktionen, die die Autonomie bei Alltagsaktivit\u00e4ten und die Lebensqualit\u00e4t steigern k\u00f6nnen. Die intrathekale Applikation ist auch bei schweren spinalen Deformit\u00e4ten unter Bildgebung in der Regel komplikationsfrei durchf\u00fchrbar. Zum Monitoring des therapeutischen Effekts ist eine regelm\u00e4\u00dfige systematische Evaluation des motorischen Status mit validierten Instrumenten zu empfehlen. Die Dokumentation in einem SMA-Register erm\u00f6glicht den systematischen Aufbau einer Datenbasis zur Weiterentwicklung des therapeutischen Gesamtkonzepts. Relevante Aspekte dieses neuartigen Therapieprinzips diskutierten Experten im Rahmen einer Konferenz in Frankfurt am Main im Februar 2019.<\/jats:p>","DOI":"10.1055\/a-0996-0994","type":"journal-article","created":{"date-parts":[[2019,12,17]],"date-time":"2019-12-17T23:44:51Z","timestamp":1576626291000},"page":"703-710","source":"Crossref","is-referenced-by-count":5,"title":["Spinale Muskelatrophie \u2013 Expertenempfehlungen zur Behandlung von erwachsenen Patienten mit Nusinersen"],"prefix":"10.1055","volume":"87","author":[{"given":"Tim","family":"Hagenacker","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr Neurologie, Universit\u00e4tsmedizin Essen"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Hermann","sequence":"additional","affiliation":[{"name":"Sektion f\u00fcr Translationale Neurodegeneration \u201eAlbrecht Kossel\u201c, Klinik und Poliklinik f\u00fcr Neurologie Universit\u00e4tsmedizin Rostock"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christoph","family":"Kamm","sequence":"additional","affiliation":[{"name":"Klinik und Poliklinik f\u00fcr Neurologie, Universit\u00e4tsmedizin Rostock"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maggie C.","family":"Walter","sequence":"additional","affiliation":[{"name":"M.A. Friedrich-Baur-Institut, Neurologische Klinik und Poliklinik, Ludwig-Maximilians-Universit\u00e4t"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Markus","family":"Weiler","sequence":"additional","affiliation":[{"name":"Neurologische Klinik, Universit\u00e4tsklinikum Heidelberg"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ren\u00e9","family":"G\u00fcnther","sequence":"additional","affiliation":[{"name":"Klinik und Poliklinik f\u00fcr Neurologie, Technische Universit\u00e4t Dresden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudia D.","family":"Wurster","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr Neurologie, Rehabilitations- und Universit\u00e4tskliniken Ulm"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christoph","family":"Kleinschnitz","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr Neurologie, Universit\u00e4tsmedizin Essen"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2019,12,17]]},"container-title":["Fortschritte der Neurologie \u00b7 Psychiatrie"],"original-title":[],"language":"de","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/a-0996-0994.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,12,18]],"date-time":"2019-12-18T11:18:58Z","timestamp":1576667938000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/a-0996-0994"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12]]},"references-count":0,"journal-issue":{"issue":"12","published-online":{"date-parts":[[2019,12,17]]},"published-print":{"date-parts":[[2019,12]]}},"URL":"https:\/\/doi.org\/10.1055\/a-0996-0994","relation":{},"ISSN":["0720-4299","1439-3522"],"issn-type":[{"value":"0720-4299","type":"print"},{"value":"1439-3522","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,12]]}}}