Casimersen (AMONDYS 45™) is an FDA-approved antisense oligonucleotide therapy for Duchenne muscular dystrophy (DMD) patients whose mutations allow exon 45 skipping. This treatment increases dystrophin production, stabilizing muscle function. Approved in 2021, Casimersen has shown promising results in the ESSENCE trial, significantly boosting dystrophin levels. While it is not a cure, this therapy offers hope for approximately 8% of DMD patients with exon 45 mutations. Clinical trials continue to assess long-term benefits and potential side effects, particularly concerning kidney health.

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This post introduces NS-089/NCNP-02, a novel exon-skipping therapy for Duchenne muscular dystrophy (DMD). Designed to target exon 44, this antisense oligonucleotide improves dystrophin restoration in DMD patients. Unlike traditional single-target ASOs, NS-089/NCNP-02 skips two different sequences within exon 44, enhancing efficiency. Studies in patient-derived cells and cynomolgus monkeys confirm its ability to induce exon skipping in both skeletal and cardiac muscle, offering a promising systemic therapy for DMD.

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