Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-... by Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K, Abbs, S, Garralda, M, Bourke, J, Wells, D, Dickson, G, Wood, M, Wilton, S, Straub, V, Kole, R, Shrewsbury, S, Sewry, C, Morgan, J, Bushby, K, Muntoni, F

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-... by Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K, Abbs, S, Garralda, M, Bourke, J, Wells, D, Dickson, G, Wood, M, Wilton, S, Straub, V, Kole, R, Shrewsbury, S, Sewry, C, Morgan, J, Bushby, K, Muntoni, F

Delivery is key: lessons learnt from developing splice-switching antisense therapies by Godfrey, C, Desviat, L, Smedsrød, B, Piétri-Rouxel, F, Denti, M, Disterer, P, Lorain, S, Nogales-Gadea, G, Sardone, V, Anwar, R, El Andaloussi, S, Lehto, T, Khoo, B, Brolin, C, , v, Goyenvalle, A, Aartsma-Rus, A, Arechavala-Gomeza, V

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities by Hammond, SM, Aartsma‐Rus, A, Alves, S, Borgos, SE, Buijsen, RAM, Collin, RWJ, Covello, G, Denti, MA, Desviat, LR, Echevarría, L, Foged, C, Gaina, G, Garanto, A, Goyenvalle, AT, Guzowska, M, Holodnuka, I, Jones, DR, Krause, S, Lehto, T, Montolio, M, Van Roon‐Mom, W, Arechavala‐Gomeza, V

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-conce... by Kinali, M, Arechavala-Gomeza, V, Feng, L, Cirak, S, Hunt, D, Adkin, C, Guglieri, M, Ashton, E, Abbs, S, Nihoyannopoulos, P, Garralda, M, Rutherford, M, McCulley, C, Popplewell, L, Graham, I, Dickson, G, Wood, M, Wells, D, Wilton, S, Kole, R, Straub, V, Bushby, K, Sewry, C, Morgan, J, Muntoni, F

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy by Straub, V, Balabanov, P, Bushby, K, Ensini, M, Goemans, N, De Luca, A, Pereda, A, Hemmings, R, Campion, G, Kaye, E, Arechavala-Gomeza, V, Goyenvalle, A, Niks, E, Veldhuizen, O, Furlong, P, Stoyanova-Beninska, V, Wood, M, Johnson, A, Mercuri, E, Muntoni, F, Sepodes, B, Haas, M, Vroom, E, Aartsma-Rus, A