Viltolarsen y la Distrofia Muscular de Duchenne
- Ignacio González Ruiz
- Sara Ortega Cubero
ISSN: 2173-0903
Year of publication: 2022
Issue: 45
Type: Article
More publications in: MoleQla: revista de Ciencias de la Universidad Pablo de Olavide
Abstract
Este fármaco consiste en un oligonucleótido antisentido de morfolino fosforodiamidato, que se emplea en el tratamiento de la distrofia muscular de Duchenne (DMD). Este compuesto se basa en la terapia de omisión del exón, permitiendo que se obtenga una proteína funcional, aunque el ARN mensajero no contenga todos los exones de la proteína normal.
Bibliographic References
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