Systemic administration of the antisense oligonucleotide <scp>NS</scp> ‐089/ <scp>NCNP</scp> ‐02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/ <scp>II</scp> clinical trial

Abstract

Exon-skipping therapy using ASOs shows promise in selected patients, and this first-in-human study is expected to provide critical information for subsequent clinical development of NS-089/NCNP-02.