Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1.

Pechmann, Astrid; Baumann, Matthias; Bernert, Günther; Flotats-Bastardas, Marina; Gruber-Sedlmayr, Ursula; von der Hagen, Maja; Hasselmann, Oswald; Hobbiebrunken, Elke; Horber, Veronka; Johannsen, Jessika; Kellersmann, Anna; Köhler, Cornelia; von Moers, Arpad; Müller-Felber, Wolfgang; Plecko, Barbara; Reihle, Christof; Schlachter, Kurt; Schreiber, Gudrun; Schwartz, Oliver; Smitka, Martin; Steiner, Elisabeth; Stoltenburg, Corinna; Stüve, Burkhard; Theophil, Manuela; Weiß, Claudia; Wiegand, Gert; Wilichowski, Ekkehard; Winter, Benedikt; Wittmann, Wolfgang; Schara, Ulrike; Kirschner, Janbernd

doi:10.3233/jnd-190441

Public

Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1.

Published in Journal of neuromuscular diseases • Jan 1, 2020

NobleIDark:/48914/w1/20260526/8E15581BRecord on NobleID

Authors:

Astrid Pechmann

,

Matthias Baumann

,

Günther Bernert

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 ch...

Discussions