A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation.

EMBO Mol Med · 2012 · PMC3494875 · PMID 23027640

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did not reach statistical significanceno p-value reported

Application of PTC124 was safe over a 48-week treatment period; however, the ambitious primary endpoint did not reach statistical significance ( http://ptct.client.shareholder.com/releasedetail.cfm?ReleaseID=518941 ).

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