Poster 147 : Six-year long-term safety and efficacy of Golodirsen in patients with DMD vs mutation-matched external controls

Muntoni F, Seferian A, Straub V, Guglieri M, Servais L, Wilk-Durakiewicz E, Ni X, Gao P, Hu M, Iff J, Hill L, Sehinovych I, Orogun L, Mercuri E

Neuromuscular disorders : NMD