Article

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

2023-02-08

Abstract excerpt

<h4>Aim</h4> The purpose of this study is to evaluate the safety and pharmacokinetics of the novel morpholino oligomer NS-089/NCNP-02 which can induce exon 44 skipping, in patients with DMD. Additionally, we aimed to identify markers predictive of therapeutic efficacy and determine the optimal dosing for future studies. <h4>Methods</h4> This is an open-label, dose-escalation, two-center phase I/II trial in ambulan...

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Literature Corpus work: 1723fb59-1d09-5c2f-9046-b4d024c5b1c8
DOI: 10.1101/2023.02.06.23285500

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