Golodirsen is a drug used to treat certain mutations that cause Duchenne muscular dystrophy (DMD). Golodirsen is a morpholino antisense oligomer designed to treat about 8% of patients with Duchenne Muscular Dystrophy (DMD).
- Mechanism
- Curator reviewed · 9 references
The hallmark of Duchenne Muscular Dystrophy is the absence of the important muscle stabilizing protein, dystrophin, that is caused by a deletion mutation on the DMD (dystrophin) gene. This results in the production of a non-functional protein. Lack of dystrophin protein leads to progressive muscle weakness and degeneration.
Golodirsen binds to exon 53 of dystrophin pre-mRNA on the DMD gene, excluding this protein coding unit during mRNA processing. The exclusion (or skipping) of exon 53 by golodirsen has the end result of changing out-of-frame mRNA to in-frame mRNA, inducing the production of dystrophin. The production of an imperfect dystrophin protein induced by golodirsen likely leads to a less severe condition, Becker Muscular Dystrophy (BMD), characterized by the production of a truncated dystrophin protein. Patients with BMD generally can expect a longer lifespan and improved quality of life.
- Primary indication
- Golodirsen is indicated to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that would benefit from exon 53 skipping.Curator reviewed · 1 structured indication
- First approval
- United States, 2019
- Code names
- NG-12-0163 · SRP-4053 · WHO 10355
- Brand names
Resolves to
What you can answer from here — as of March 06, 2025
Which drugs share a target with Golodirsen, and which of those have an active Phase 3 trial?
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