Translarna could be available in weeks for English children with Duchenne
The National Institute for Health and Care Excellence (NICE), the medicines cost-effectiveness watchdog for England and Wales, has recommended Translarna (ataluren), from PTC Therapeutics (Nasdaq: PTCT), for children with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation, after reaching a ‘managed access agreement’.
This agreement established financial and clinical details surrounding the use of the treatment and was a condition for making the drug available imposed by NICE in its April draft guidance.
The share price of PTC Therapeutics, a US pharma company, rose by 8% on Wednesday, when the news was announced.
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