Eteplirsen
This RNA-based therapy is designed to help the body skip over a specific faulty section of the dystrophin gene so that a shorter but partially functional protein can be made. It is used to treat Duchenne muscular dystrophy in patients with a specific mutation.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
HBD / HBA
- / -
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
Sıkça Sorulan Sorular
This RNA-based therapy is designed to help the body skip over a specific faulty section of the dystrophin gene so that a shorter but partially functional protein can be made. It is used to treat Duchenne muscular dystrophy in patients with a specific mutation.
Yes, Eteplirsen is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL2108278. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
Embed This Widget
Add the script tag and a data attribute to embed this widget.
<script src="https://cdn.jsdelivr.net/npm/drugfyi-embed@1/dist/embed.min.js" defer></script>
<div data-drugfyi="drug" data-slug="eteplirsen"></div>Embed via iframe for maximum compatibility.
<iframe src="https://drugfyi.com/iframe/drug/eteplirsen/" width="420" height="400" frameborder="0" style="border:0;border-radius:10px;max-width:100%" loading="lazy"></iframe>Paste this URL in WordPress, Medium, or any oEmbed-compatible platform.
https://drugfyi.com/drug/eteplirsen/Add a dynamic SVG badge to your README or docs.
[](https://drugfyi.com/drug/eteplirsen/)Use the native HTML custom element.
<script src="https://cdn.jsdelivr.net/npm/drugfyi-embed@1/dist/embed.min.js" defer></script>
<drugfyi-drug slug="eteplirsen"></drugfyi-drug>