PLATELET DYSFUNCTION AND MDS FEATURES DUE TO NOVEL SOMATIC GATA1 MUTATION حسب Freson, K, de Waele, L, Vandenberghe, P, de Vos, R, Vyas, P, van Geet, C

A Myelodysplastic Syndrome Associated with a Clonal GATA1 Mutation. حسب Freson, K, De Waele, L, Thys, C, Wittevrongel, C, Vandenberghe, P, De Vos, R, Vyas, P, Van Geet, C

Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy حسب Servais, L, Straathof, CSM, Schara, U, Klein, A, Leinonen, M, Hasham, S, Meier, T, De Waele, L, Gordish-Dressman, H, McDonald, CM, Mayer, OH, Voit, T, Mercuri, E, Buyse, GM

Patients’ Perceptions of Nusinersen Effects According to Their Responder Status حسب Lilien, C, Vrscaj, E, Thapaliya, G, Deconinck, N, De Waele, L, Duong, T, Haberlová, J, Kumhera, M, Peirens, G, Szabo, L, Tahon, V, Tang, WJ, Benmhammed, N, Médard, L, Servais, L

Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study حسب Chabanon, A, Seferian, AM, Daron, A, Péréon, Y, Cances, C, Vuillerot, C, De Waele, L, Cuisset, J-M, Laugel, V, Schara, U, Gidaro, T, Gilabert, S, Hogrel, J-Y, Baudin, P-Y, Carlier, P, Fournier, E, Lowes, LP, Hellbach, N, Seabrook, T, Toledano, E, Annoussamy, M, Servais, L

ANO5 Gene Analysis in a Large Cohort of Patients with Anoctaminopathy: Confirmation of Male Prevalence and High Occurrence of the Common Exon 5 Gene Mutation حسب Sarkozy, A, Hicks, D, Hudson, J, Laval, S, Barresi, R, Hilton-Jones, D, Deschauer, M, Harris, E, Rufibach, L, Hwang, E, Bashir, R, Walter, M, Krause, S, van den Bergh, P, Illa, I, Pénisson-Besnier, I, De Waele, L, Turnbull, D, Guglieri, M, Schrank, B, Schoser, B, Seeger, J, Schreiber, H, Gläser, D, Eagle, M

ANO5 gene analysis in a large cohort of patients with anoctaminopathy: confirmation of male prevalence and high occurrence of the common exon 5 gene mutation. حسب Sarkozy, A, Hicks, D, Hudson, J, Laval, S, Barresi, R, Hilton-Jones, D, Deschauer, M, Harris, E, Rufibach, L, Hwang, E, Bashir, R, Walter, M, Krause, S, van den Bergh, P, Illa, I, Pénisson-Besnier, I, De Waele, L, Turnbull, D, Guglieri, M, Schrank, B, Schoser, B, Seeger, J, Schreiber, H, Gläser, D, Eagle, M