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

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy by Straub, V, Balabanov, P, Bushby, K, Ensini, M, Goemans, N, De Luca, A, Pereda, A, Hemmings, R, Campion, G, Kaye, E, Arechavala-Gomeza, V, Goyenvalle, A, Niks, E, Veldhuizen, O, Furlong, P, Stoyanova-Beninska, V, Wood, M, Johnson, A, Mercuri, E, Muntoni, F, Sepodes, B, Haas, M, Vroom, E, Aartsma-Rus, A