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# PTC Therapeutics Completes NDA Submission for Duchenne
- URL: https://www.fdaweb.com/ptc-therapeutics-completes-nda-submission-for-duchenne/
- Published: 2016-01-08T12:00:00.000Z
- Updated: 2026-09-15T02:26:04.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5134390

PTC Therapeutics has completed its rolling submission to FDA of an NDA for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for treating nonsense mutation Duchenne muscular dystrophy (DMD). According to the company, the therapy is designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. This mutation or alteration in the genetic code that prematurely halts the synthesis of an essential protein, results in a disorder determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in DMD, says the company.