Eteplirsen
Eteplirsen
Eteplirsen (pronounced eh-TEP-lihr-sen) is a pharmaceutical drug used in the treatment of Duchenne muscular dystrophy (DMD). It is marketed under the brand name Exondys 51 by Sarepta Therapeutics.
Etymology
The name "Eteplirsen" is derived from the drug's chemical structure. The "Etep" part of the name is an abbreviation of "Eteplirsen", while the "lirsen" part is a common suffix used in the naming of antisense oligonucleotides, a class of drugs to which Eteplirsen belongs.
Mechanism of Action
Eteplirsen works by binding to a specific section of the DMD gene, causing the cell's machinery to skip over a faulty part of the gene when it is being read. This allows a functional, though truncated, dystrophin protein to be produced, which is essential for muscle function.
Usage
Eteplirsen is used in the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. The drug is administered via intravenous infusion.
Side Effects
Common side effects of Eteplirsen include balance disorder, vomiting, and contact dermatitis. Serious side effects may include kidney damage and low platelet counts.
Related Terms
- Duchenne muscular dystrophy
- Sarepta Therapeutics
- Antisense oligonucleotides
- DMD gene
- Dystrophin
- Intravenous infusion
External links
- Medical encyclopedia article on Eteplirsen
- Wikipedia's article - Eteplirsen
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