Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy 著者: 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

Prognostic value of different anthropometric indices over different measurement intervals to predict mortality in 6–59-month-old children 著者: Briend, A, Myatt, M, Berkley, JA, Black, RE, Boyd, E, Garenne, M, Lelijveld, N, Isanaka, S, McDonald, CM, Mwangwome, M, O'Brien, KS, Schwinger, C, Stobaugh, H, Taneja, S, West, KP, Khara, T

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy 著者: 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

Anthropometric deficits and the associated risk of death by age and sex in children aged 6-59 months: a meta-analysis 著者: Thurstans, S, Wrottesley, SV, Fenn, B, Khara, T, Bahwere, P, Berkley, JA, Black, RE, Boyd, E, Garenne, M, Isanaka, S, Lelijveld, N, McDonald, CM, Mertens, A, Mwangome, M, O'Brien, KS, Stobaugh, H, Taneja, S, West, KP, Guerrero, S, Kerac, M, Briend, A, Myatt, M

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy 著者: 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)