Overview of viltolarsen (NS-065/NCNP-01) clinical trials

Trial publications Phase I open-label trial (NCT02081625) Exploratory Study of NS-065/NCNP-01 in DMD Komaki H, Nagata T, Saito T, et al. Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy. Science translational medicine 2018;10 Shimizu R, Ohata M, Tachimori H, et al. Expectations and anxieties of […]