WVE-N531 yields 53% mean exon 53 skipping in skeletal muscle of boys with Duchenne muscular dystrophy (DMD) after three biweekly doses

Tillinger, M; Lake, S; Servais, L; Campbell, C; Xu, X; Hart, A; Haegele, J; Singh, K; Rheinhardt, J; Ghosh, A; Xu, D; Panzara, M; Li-Kwai-Cheung, A

NEUROMUSCULAR DISORDERS, 2023; 33 (): S102