The findings, detailed by the National Center of Neurology and Psychiatry, stem from an open-label trial involving six participants receiving weekly intravenous doses. Over the five-year observation period, the study reported no serious adverse events or treatment-related anaphylaxis, with no patients discontinuing the regimen. Clinical assessments revealed that participants maintained or improved motor function, including upper limb stability in those who eventually became non-ambulant. When measured against natural history data for the disease, the functional outcomes showed a favorable trajectory for patients treated with the antisense oligonucleotide.

NS Pharma President Yukiteru Sugiyama noted that these longer-term results underscore the potential of the therapy to provide meaningful options for patients facing the progressive muscle weakness associated with the condition. As the extension study continues, Nippon Shinyaku and NS Pharma are also conducting a global Phase II trial to further validate these outcomes. Duchenne muscular dystrophy remains a severe condition characterized by the loss of skeletal, cardiac, and respiratory muscle, often necessitating wheelchair use by adolescence. Researchers hope that continued investigation will clarify the durability of these early clinical improvements.