Exon-skipping therapy aims to restore dystrophin production in Duchenne muscular dystrophy (DMD) by using antisense oligonucleotides (AONs) to bypass mutations. Eteplirsen and viltolarsen target exon 51, producing a truncated but functional dystrophin protein. While promising, challenges remain, including low dystrophin levels and variable patient responses. Gene therapy and stop codon read-through drugs offer alternative strategies. Early intervention and combination therapies may improve outcomes for DMD patients.

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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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