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Scots Hammer PTC's Translarna As EU Reviews Conditional Approval

This article was originally published in Scrip

Executive Summary

In another blow for PTC Therapeutics' orphan drug for Duchenne muscular dystrophy, Translarna (ataluren), the Scottish Medicines Consortium has rejected the use of the product on the national health service, saying that the company had failed to justify its high cost in relation to its clinical benefits. The move comes in wake of the US Food and Drug Administration's February refusal to accept PTC's filing for Translarna.

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