Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence.

Authors: Weiß C, Vill K, Baumann M, Bernert G, Blaschek A, Eisenkölbl A, Flotats-Bastardas M, Friese J, Ganter C, Goldhahn K, Hahn A, von der Hagen M, Hartmann H, Hasselmann O, Horber V, Husain RA, Illsinger S, Jacquier D, Johannsen J, Köhler C, Kölbel H, Kolodzig M, Klein A, Pechmann A, von Moers A, Müller-Felber W, Rauscher C, Schara-Schmidt U, Schreiber G, Schwartz O, Sproß J, Stettner GM, Stoltenburg C, Stumpe E, Trollmann R, Wiegand G, Wilichowski E, Kirschner J, Ziegler A, collaborators and members of the INTEGRATE ATMP consortium

Journal: Journal of neuromuscular diseases