What is the role of ataluren (PTC124) in the management of muscular dystrophy?

Updated: Aug 17, 2020
  • Author: Twee T Do, MD; Chief Editor: Jeffrey D Thomson, MD  more...
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Answer

Ataluren (formerly known as PTC124; PTC Therapeutics, Inc, South Plainfield, NJ) is an oxadiazole compound that, when taken orally, can override nonsense stop translation signals induced by the dystrophin gene mutation; the protein produced is thus the full-length protein. [47, 48] In Europe, it is used to treat Duchenne MD patients who have a nonsense mutation in the dystrophin gene, can walk, and are older than 5 years. It is not approved by the US Food and Drug Administration for this purpose.


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