Clinical evidence supporting early treatment of patients with spinal muscular atrophy: Current perspectives by Dangouloff, T, Servais, LJP

Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients by Catapano, F, Domingos, J, Perry, M, Ricotti, V, Phillips, L, Servais, LJP, Seferian, A, de Groot, I, Krom, YD, Niks, EH, Verschuuren, JJ, Straub, V, Voit, T, Morgan, J, Muntoni, F

Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort study by Aragon-Gawinska, K, Seferian, AM, Daron, A, Gargaun, E, Vuillerot, C, Cances, C, Ropars, J, Chouchane, M, Cuppen, I, Hughes, I, Illingworth, M, Marini-Bettolo, C, Rambaud, J, Taytard, J, Annoussamy, M, Scoto, M, Gidaro, T, Servais, LJP

ASC-1 is a cell cycle regulator associated with severe and mild forms of myopathy by Villar-Quiles, RN, Catervi, F, Cabet, E, Juntas-Morales, R, Genetti, CA, Gidaro, T, Koparir, A, Yüksel, A, Coppens, S, Deconinck, N, Pierce-Hoffman, E, Lornage, X, Durigneux, J, Laporte, J, Rendu, J, Romero, NB, Beggs, AH, Servais, LJP, Cossée, M, Olivé, M, Böhm, J, Duband-Goulet, I, Ferreiro, A

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy by Conrado, DJ, Larkindale, J, Berg, A, Hill, M, Burton, J, Abrams, KR, Abresch, RT, Bronson, A, Chapman, D, Crowther, M, Duong, T, Gordish-Dressman, H, Harnisch, L, Henricson, E, Kim, S, McDonald, CM, Schmidt, S, Vong, C, Wang, X, Wong, BL, Yong, F, Romero, K, Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC)

Other Authors: “…Servais, LJP…”

Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM by Klingels, K, Mayhew, AG, Mazzone, ES, Duong, T, Decostre, V, Werlauff, U, Vroom, E, Mercuri, E, Goemans, NM, Upper Limb Clinical Outcome Group

Other Authors: “…Servais, LJP…”