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Kirschner, Janbernd; Bernert, Günther; Butoianu, Nina; De Waele, Liesbeth; Fattal-Valevski, Aviva; Haberlova, Jana; Moreno, Teresa; Klein, Andrea; Kostera-Pruszczyk, Anna; Mercuri, Eugenio; Quijano-Roy, Susana; Sejersen, Thomas; Tizzano, Eduardo F; van der Pol, W Ludo; Wallace, Sean; Zafeiriou, Dimitrios; Ziegler, Andreas; Muntoni, Francesco; Servais, Laurent (2024). 2024 update: European consensus statement on gene therapy for spinal muscular atrophy. European journal of paediatric neurology, 51, pp. 73-78. Elsevier 10.1016/j.ejpn.2024.06.001
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
