.Pharmacolibrary.Drugs.ATC.M.M09AX08 .Pharmacolibrary.Drugs.ATC.M.M09AX08| name: | Golodirsen |
| ATC code: | M09AX08 | route: | intravenous |
| n-compartments | 2 |
Golodirsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. It is approved for use in the United States.
Pharmacokinetics in male pediatric patients with DMD (aged 7–13 years) following intravenous administration.
Goey, AKL, et al., & East, L (2024). Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy. Drug metabolism and disposition: the biological fate of chemicals 52(12) 1396–1406. DOI:10.1124/dmd.124.001819 PUBMED:https://pubmed.ncbi.nlm.nih.gov/39516029