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Phase 2 Study of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping
jRCT2041250028Phase 2Active, not recruiting
- Sponsor
- Nippon Shinyaku
- Start
- May 2025
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
Phase 2, open-label trial of brogidirsen in Duchenne muscular dystrophy (DMD) (2 cohorts), sponsored by Nippon Shinyaku. Primary endpoints: Adverse events and adverse drug reactions, Plasma pharmacokinetic parameters of NS-089/NCNP-02 and 2 more. Started May 2025; status: active, not recruiting.
02
Drugs
brogidirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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