CAS: 1143516-05-5
Purpose: Indicated for patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. By promoting exon skipping during pre-mRNA splicing, it restores partial dystrophin production, helping to slow muscle degeneration and delay disease progression. It is not a curative therapy and must be administered by intravenous infusion.
Formula: C4H12N2O





