Families' anger as Duchenne boys denied 'breakthrough' drug

NICE has announced it won't be recommending funding on the NHS for the drug Translarna, which could keep boys with life-limiting Duchenne muscular dystrophy walking for longer.

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Families 'devastated' by NICE recommendation on drug

A group of parents have told of the devastation they felt at having to tell their children that they still can't have a drug that could dramatically improve their lives.

Harry Barnley from Aldershot and Jagger Curtis from Romsey suffer from the life limiting disease, Duchenne muscular dystrophy.

It causes the muscles to weaken over time and sees most young boys in wheelchairs by the age of 11.

But if used early enough, it's been shown that the drug 'Translarna' could help delay the muscle wasting process in some children and keep them on their feet for longer.

The drug can cost tens of thousands of pounds a year and today the National Institute for Health and Care Excellence (NICE) said there wasn't yet enough evidence to support the benefits.

Watch Emma Wilkinson's full report:

Muscular dystrophy charity will 'keep on fighting'

Muscular Dystrophy UK says a decision by the National Institute of Health and Care Excellence (NICE) not to recommend funding for a drug to treat Duchenne muscular dystrophy is a 'huge blow'.

The charity said families of those with the muscle-wasting condition have been waiting for over a year for a decision.

The drug, Translarna, is available in other EU countries and the company which makes it says trials show it is beneficial.

It can only be used by children whose muscular dystrophy is caused by what's known as a nonsense mutation in the dystrophin gene.

NICE said today it has asked for further clarification from PCT Therapeutics on the size of the benefit of the drug. It said it needs the company to provide the results of a confirmatory study of as soon as possible, as well as further justification for the cost of the drug.

Nic Bungay from Muscular Dystrophy UK says it isn't the end of the road:

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Duchenne boys won't get 'breakthrough' drug on NHS

Campaigners with David Cameron earlier this year Credit: ITV Meridian

After months of delays, and despite a prominent campaign, NICE has announced it will not recommend funding on the NHS for breakthrough drug Translarna which could keep boys with life-limiting Duchenne muscular dystrophy walking for longer.

Ahead of confirming its final position in the coming months, NICE have asked the company to provide further justification for the cost of the drug.

Boys affected by the condition deliver appeal letters to Number 10 Credit: ITV Meridian

In August 2014, Translarna became the first drug addressing the cause of Duchenne muscular dystrophy to be approved in the EU. It has been available in several EU countries since last year.

Archie Hill, 10, from Buckinghamshire delivers a petition to Downing Street Credit: ITV Meridian

Families of the boys affected have spoken of their anger at the news.

“We are absolutely devastated. How do we tell Archie he is not allowed a drug that will keep him walking and living for longer because NHS England and drug companies cannot agree on a price.

– Louisa Hill, mother of Archie, from Buckinghamshire
Louisa Hills from Buckinghamshire Credit: ITV Meridian

“Today's news is extremely disappointing, and a bitter blow for all of us awaiting the news. Having spoken to some of the families affected, it really is impossible to describe their heartbreak, particularly as just last week Lothian NHS Trust approved the drug for a family in Scotland.

“Many parents across the rest of the UK are watching their child weaken and lose mobility day by day, as this really is a race against time. If we are not careful, it will be too late for many of these boys to even be eligible to take Translarna."

– Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK
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