NICE grants access to first Duchenne muscular dystrophy drug for children

Steve McCabe MP has welcomed news that NICE have approved access to a drug which tackles the underlying causes of life-limiting Duchenne muscular dystrophy, a condition that affects children.

For the first time ever, children will be able to access a drug tackling an underlying cause of life-limiting, Duchenne muscular dystrophy through NHS England. Translarna could keep children with the severe muscle-wasting condition walking for longer.

In August 2014, Translarna became the first drug addressing a genetic cause of Duchenne muscular dystrophy to be approved in the EU. It has been available to families in a number of European countries for over a year. Duchenne muscular dystrophy almost always affects boys.

The families of just over 50 eligible children in England – 60 across the UK – have waited almost 18 months for a decision from NHS England on funding for the drug. The new Agreement is intended to cover access to Translarna for eligible children in England for 5 years. During this time, 50 children will be closely monitored and their progress compared to that of other boys with the condition. The data gathered will inform NICE’s ultimate decision on funding the drug, when the Agreement concludes.

The news comes as a victory for families who have campaigned tirelessly with Muscular Dystrophy UK.

Duchenne muscular dystrophy affects 2,500 people in the UK. The condition causes muscles to weaken and waste over time, leading to increasing disability. It affects the heart and muscles used for breathing, cutting lives short.

Muscular Dystrophy UK is the charity for 70,000 children and adults living with muscle-wasting conditions. They provide vital information, advice and support to help people live as independently as possible. They also work to accelerate progress in research and drive the campaign for access to emerging treatments.

“I have worked with Muscular Dystrophy UK and have been campaigning with my colleagues in Parliament for access to Translarna so this is very welcome news for families who are affected by Duchenne muscular dystrophy. I think that this is an historic moment and a major step forward in the fight for potential treatments for Duchenne muscular dystrophy.”

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