Category: Genetic Disorders

Eteplirsen Overview

Eteplirsen (brand name Exondys 51) is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Eteplirsen only targets specific mutations and can be used to treat about 14% of DMD cases.[1][2] Eteplirsen is a form of antisense therapy. Eteplirsen was developed by Sarepta Therapeutics. After a controversial debate surrounding the drug's efficacy, during which two FDA review panel members resigned in pro...

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Possible Dosages for this and Related Drugs:

  • Solution: 100mg/2ml (50mg/ml), 500mg/10ml (50mg/ml)
Note: Above list includes dosages for all drugs with the same combination of active ingredients.

Other drugs which contain Eteplirsen or a similar ingredient: (1 result)