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Golodirsen is a phosphodiesteric morpholino oligomer (PMO) that specifically targets exon 53 of the pre-protein mRNA of myotonic dystrophy protein culture proteins, and can be used in studies related to Duchenne muscular dystrophy (DMD).
| Molecular Weight | 8647.00 |
| CAS Number | 1422959-91-8 |
| Form | Solid |
| Solubility (25°C) | Water 100 mg/mL |
| Storage | -20°C, dry |
[2] S Anwar et al. Drugs Today (Barc). Golodirsen for Duchenne muscular dystrophy
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