Abstract
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with dmd due to mutations amenable to exon 44 skipping. participants will receive a selected dose of ns-089 ncnp-02 administered once weekly. the study consists of 2 parts: part 1 and part 2. six participants cohort 1 will participate in both part 1 and part 2 and 14 participants cohort 2 will be added for part 2.>
