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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
Published 2016Journal article -
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Nusinersen versus sham control in later-onset spinal muscular atrophy by Mercuri, E, Darras, BT, Chiriboga, CA, Day, JW, Campbell, C, Connolly, AM, Iannaccone, ST, Kirschner, J, Kuntz, NL, Saito, K, Shieh, PB, Tulinius, M, Mazzone, ES, Montes, J, Bishop, KM, Yang, Q, Foster, R, Gheuens, S, Bennett, CF, Farwell, W, Schneider, E, De Vivo, DC, Finkel, RS
Published 2018Journal article