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Kirschner, Janbernd; Bernert, Günther; v. der Hagen, Maja; Hahn, Andreas; Johannsen, Jessika; Klein, Andrea; Müller-Felber, Wolfgang; Ziegler, Andreas; Schara, Ulrike (2020). Gene therapy for spinal muscular atrophy with onasemnogene abeparvovec. Statement of the German Speaking Society of Neuropediatrics. Monatsschrift Kinderheilkunde, 168(10), pp. 938-941. Springer-Verlag 10.1007/s00112-020-00944-6
Servais, Laurent; Straathof, Chiara S M; Schara, Ulrike; Klein, Andrea; Leinonen, Mika; Hasham, Shabir; Meier, Thomas; De Waele, Liesbeth; Gordish-Dressman, Heather; McDonald, Craig M; Mayer, Oscar H; Voit, Thomas; Mercuri, Eugenio; Buyse, Gunnar M (2020). Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy. Neuromuscular disorders, 30(1), pp. 5-16. Elsevier 10.1016/j.nmd.2019.10.008
