Brogidirsen Shows 5-Year Motor Function Benefit in DMD Trial
Paramus, N.J. – October 01, 2026 -- NS Pharma, Inc. reported 5-year efficacy and safety data showing its investigational therapy brogidirsen maintained or improved motor function in Duchenne muscular dystrophy (DMD) patients amenable to exon 44 skipping, with zero treatment discontinuations across the study period.
Six-participant trial records no serious adverse events over five years
The data, presented by Japan's National Center of Neurology and Psychiatry (NCNP) at the 31st annual International Congress of the World Muscle Society in Hiroshima, Japan, covered an open-label extension of an investigator-initiated clinical trial. All six participants received weekly intravenous dosing of brogidirsen (NS-089/NCNP-02), an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP. After five years of administration, no serious or severe adverse events, anaphylaxis, or discontinuations tied to long-term dosing were recorded.