A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy by Victor, RG, Sweeney, HL, Finkel, R, McDonald, CM, Byrne, B, Eagle, M, Goemans, N, Vandenborne, K, Dubrovsky, AL, Topaloglu, H, Miceli, MC, Furlong, P, Landry, J, Elashoff, R, Tadalafil DMD Study Group, Servais, L

A Comparative Study of α-Dystroglycan Glycosylation in Dystroglycanopathies Suggests that the Hypoglycosylation of α-Dystroglycan Does Not Consistently Correlate with Clinical Seve... by Jimenez-Mallebrera, C, Torelli, S, Feng, L, Kim, J, Godfrey, C, Clement, E, Mein, R, Abbs, S, Brown, S, Campbell, K, Kröger, S, Talim, B, Topaloglu, H, Quinlivan, R, Roper, H, Childs, A, Kinali, M, Sewry, C, Muntoni, F

Refining genotype phenotype correlations in muscular dystrophies with defective glycosylation of dystroglycan by Godfrey, C, Clement, E, Mein, R, Brockington, M, Smith, J, Talim, B, Straub, V, Robb, S, Quinlivan, R, Feng, L, Jimenez-Mallebrera, C, Mercuri, E, Manzur, A, Kinali, M, Torelli, S, Brown, S, Sewry, C, Bushby, K, Topaloglu, H, North, K, Abbs, S, Muntoni, F

Brain involvement in muscular dystrophies with defective dystroglycan glycosylation by Clement, E, Mercuri, E, Godfrey, C, Smith, J, Robb, S, Kinali, M, Straub, V, Bushby, K, Manzur, A, Talim, B, Cowan, F, Quinlivan, R, Klein, A, Longman, C, McWilliam, R, Topaloglu, H, Mein, R, Abbs, S, North, K, Barkovich, A, Rutherford, M, Muntoni, F

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy by Finkel, RS, Mercuri, E, Darras, BT, Connolly, AM, Kuntz, NL, Kirschner, J, Chiriboga, CA, Saito, K, Laurent Servais, Tizzano, E, Topaloglu, H, Tulinius, M, Montes, J, Glanzman, AG, Bishop, K, Zhong, ZJ, Gheuens, S, Bennett, CF, Schneider, E, Farwell, W, De Vivo, DC, ENDEAR Study Group