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Duchenne Muscular Dystrophy - Translarna (ATALUREN) Significantly Preserves Ability to Walk for Longer

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4 February 2020

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. 

Boys with nonsense mutation treated with translarna preserved the ability to walk for years longer than those without the drug.

Currently, translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older.

To read more: http://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-first-publication-real-world-data