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NICE Recommends Translarna

Positive news for Translarna today.

http://ir.ptcbio.com/releasedetail.cfm?ReleaseID=965331

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Permalink Reply by Keith & Jill VE on April 15, 2016 at 11:54am

This is great news!

Forgive my ignorance, but would this help boys without a nonsense mutation, deletions for example? Also, any possibility to have the product shipped to the US, like deflazacort?

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Permalink Reply by Jay Griffin on April 15, 2016 at 11:58am

If I am not mistaken, it's exon 51... This could not be shipped to the US.. My son deletions 51-55 (not eligible even it was approved in the US. I am very happy for them. This approval gives great momentum for the DMD community around the world

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Permalink Reply by Aleksandar on April 15, 2016 at 2:26pm

Translarna is for nonsense mutation. This approval is even more frustrating to US citizens. Nice always seemed stricter as far as New Drugs are approved then rest of the world, yet they approve Translarna, but FDA does not even warrant a review. GREAT JOB FDA, YOU ROCK AT HELPING THE KIDS. (PS. I am very happy for kids in UK, I'm just fed up with FDA).

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Permalink Reply by Nida Zuberi on April 15, 2016 at 3:40pm

This may help push FDA to reconsider. PTC is now resubmitting their file to the Canadian health organization too. Let's see if they approve it. Is there anything we can do to push for a FDA approval. Isn't time of the essence here.