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    Has all of the relevant evidence been taken into account?

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    Are the summaries of the criteria considered by the committee, and the clinical and economic considerations reasonable interpretations of the evidence?

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    Are the provisional recommendations sound and a suitable basis for guidance on the use of ataluren in the context of national commissioning by NHS England?

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Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (review of HST3)

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2 Information about ataluren

Marketing authorisation indication

2.1 Ataluren (Translarna, PTC Therapeutics) has a conditional marketing authorisation for 'the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older'.

Dosage in the marketing authorisation

2.2 The dosage schedule is available in the summary of product characteristics.

Price

2.3 The price for ataluren is £2,532 per box of thirty 125 mg sachets, £5,064 per box of thirty 250 mg sachets and £20,256 per box of thirty 1,000 mg sachets (excluding VAT; BNF online accessed September 2022).

The company has a commercial arrangement, which would have applied if the technology had been recommended.