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PTC Therapeutics begins rolling submission of Translarna's NDA to the US FDA for treating nonsense mutation Duchenne muscular dystrophy.

M2 PHARMA-December 24, 2014-PTC Therapeutics begins rolling submission of Translarna's NDA to the US FDA for treating nonsense mutation Duchenne muscular dystrophy

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Biopharmaceutical company PTC Therapeutics (NasdaqGS:PTCT) reported on Tuesday the initiation of a rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

The company said a rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis.

This new drug application for Translarna is expected to be finalised in Q4 2015 upon completion of the ACT DMD confirmatory Phase 3 clinical trial.

According to the company, Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy.

Primarily affecting males, Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm and heart muscles.

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Publication:M2 Pharma
Date:Dec 24, 2014
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