Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial.
Takami IshizukaHirofumi KomakiYoshitsugu AokiHarumasa NakamuraNorio MotohashiEri TakeshitaYuko Shimizu-MotohashiAkihiko IshiyamaChihiro YoneeShinsuke MaruyamaEisuke HidaYoshitsugu AokiPublished in: Neuropsychopharmacology reports (2023)
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.