15 April 2016 - NICE has today recommended ataluren (Translarna, PTC Therapeutics) for treating children aged 5 and over with Duchenne muscular dystrophy caused by a nonsense mutation.

The drug has been called a ‘step change’ in the management of the disease which causes progressive muscle wasting and is usually fatal by age 30.

Children with the disease typically become wheelchair dependent by age 12 and the committee agreed that ataluren had the potential to delay the loss of ability to walk, one of the most important factors for patients and their families.

For more details, go to: https://www.nice.org.uk/news/press-and-media/nice-recommends-ataluren-for-treating-duchenne-muscular-dystrophy-caused-by-a-nonsense-mutation