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Pechmann, A; Baumann, M; Bernert, G; Flotats-Bastardas, M; Gruber-Sedlmayr, U; von der Hagen, M; Hasselmann, O; Hobbiebrunken, E; Horber, V; Johannsen, J; Kellersmann, A; Köhler, C; von Moers, A; Müller-Felber, W; Plecko, B; Reihle, C; Schlachter, K; Schreiber, G; Schwartz, O; Smitka, M; Steiner, E; Stoltenburg, C; Stüve, B; Theophil, M; Weiß, C; Wiegand, G; Wilichowski, E; Winter, B; Wittmann, W; Schara, U; Kirschner, J.
Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1.
J Neuromuscul Dis. 2020; 7(1): 41-46. Doi: 10.3233/JND-190441
Web of Science PubMed FullText FullText_MUG

Co-authors Med Uni Graz: Gruber-Sedlmayr Ursula; Plecko Barbara
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Abstract:: The natural history of patients with spinal muscular atrophy (SMA) has changed due to advances in standard care and development of targeted treatments. Nusinersen was the first drug approved for the treatment of all SMA patients. The transfer of clinical trial data into a real-life environment is challenging, especially regarding the advice of patients and families to what extent they can expect a benefit from the novel treatment. We report the results of a modified Delphi consensus process among child neurologists from Germany, Austria and Switzerland about the indication or continuation of nusinersen treatment in children with SMA type 1 based on different clinical case scenarios.