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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
Goyenvalle, Aurelie; Griffith, Graziella; Babbs, Arran; Andaloussi, Samir El; Ezzat, Kariem; Avril, Aurelie; Dugovic, Branislav; Chaussenot, Remi; Ferry, Arnaud; Voit, Thomas; Amthor, Helge; Bühr, Claudia; Schürch, Stefan; Wood, Matthew J. A.; Davies, Kay E.; Vaillend, Cyrille; Leumann, Christian; Garcia, Luis (2015). Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. Nature medicine, 21(3), pp. 270-275. Nature Publishing Group 10.1038/nm.3765
Brockington, Martin; Yuva, Yeliz; Prandini, Paola; Brown, Susan C.; Torelli, Silvia; Benson, Matthew A.; Herrmann, Ralf; Anderson, Louise V.B.; Bashir, Rumaisa; Burgunder, Jean-Marc; Fallet, Shari; Romero, Norma; Fardeau, Michel; Straub, Volker; Storey, Gillian; Pollitt, Christine; Richard, Isabelle; Sewry, Caroline A.; Bushby, Kate; Voit, Thomas; ... (2001). Mutations in the fukutin-related protein gene (FKRP) identify limb girdle muscular dystrophy 2I as a milder allelic variant of congenital muscular dystrophy MDC1C. Human molecular genetics, 10(25), pp. 2851-2859. Oxford University Press 10.1093/hmg/10.25.2851
Robin, Valerie; Ittig, Damian; Voit, Thomas; Leumann, Christian J.; Garcia, Luis (22 October 2012). SMN rescue by using oligonucleotides of tricyclo-DNA to induce exon 7 inclusion in SMN2 mRNA. Human gene therapy, 23(10), A37-A38. Mary Ann Liebert Inc.
