Novel therapies for spinal muscular atrophy are likely changing the patient phenotype

Spine J. 2020 Dec;20(12):1893-1898. doi: 10.1016/j.spinee.2020.08.013. Epub 2020 Aug 25.

¹ Center for Musculoskeletal Surgery Berlin Mitte; Charité University Medicine Berlin, Charitéplatz 1, Berlin 10117, Germany. Electronic address: tu-lan.vu-han@charite.de.
² Center for chronically sick children, Department of Neuropediatrics; Charité University, Medicine Berlin, Augustenburger Platz 1, Berlin 13353, Germany.
³ Center for Musculoskeletal Surgery Berlin Mitte; Charité University Medicine Berlin, Charitéplatz 1, Berlin 10117, Germany.

No abstract available

Keywords: Abeparvovec-xioi; Nusinersen; SMN; Scoliosis; Spinal musclular atrophy; Zolgensma.