Golodirsen
Golodirsen (pronounced go-lo-DIR-sen) is a medication used in the treatment of Duchenne muscular dystrophy (DMD) in certain patients. It is marketed under the brand name Vyondys 53 by Sarepta Therapeutics.
Etymology
The name "Golodirsen" is derived from the drug's chemical structure. It is a morpholino antisense oligomer that induces exon 53 skipping in the dystrophin gene.
Pharmacology
Golodirsen works by binding to exon 53 of the dystrophin pre-mRNA, resulting in exclusion, or "skipping," of this exon during mRNA processing. Skipping exon 53 allows for the production of an internally truncated, but functional form of dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
Usage
Golodirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This group represents about 8% of the total DMD population.
Side Effects
Common side effects of Golodirsen include headache, fever, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea. Serious side effects may include hypersensitivity reactions and kidney damage.
Related Terms
- Duchenne muscular dystrophy
- Sarepta Therapeutics
- Vyondys 53
- Morpholino
- Antisense oligomer
- Exon skipping
External links
- Medical encyclopedia article on Golodirsen
- Wikipedia's article - Golodirsen
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