Families battle for access to Translarna

Published Date
08/12/2014
Author
Peter Sutton
Category
Campaigns
Access to Translarna

The Muscular Dystrophy Campaign is joining forces with families affected by Duchenne muscular dystrophy to lead the fight for fast access to a new treatment, Translarna.

Families learned last week that NHS England is putting on hold its decision on Translarna, following the threat of legal action over the way it decides which new treatments to fund.

Translarna would be made available to boys whose Duchenne muscular dystrophy is caused by a nonsense mutation, who are over 5 and who can still walk.

Because of this criteria, families ‘face a race against time’ to access Translarna before their son loses the ability to walk. NHS England hopes to restart the assessment of Translarna in the New Year, but so far no firm commitments have been made.

The Muscular Dystrophy Campaign is calling for NHS England to take emergency steps to ensure that the drug is made available as a matter of urgency, to the children it could help, by April 2015.

Archie (left), with his big brother, Leyton

Louisa Hill’s son, Archie, is eligible for Translarna. Alongside the Muscular Dystrophy Campaign, she will tomorrow be taking the fight for fast access to Translarna to the Health Minister, George Freeman MP. Louisa said:

“People making these decisions (on whether to fund a treatment) don’t understand the impact on children of even a small change. It gives them more time to run and play football with their friends. It’s really buying precious time. Archie will have to deal with very difficult mental and physical challenges as his condition progresses. Translarna is buying time for Archie just to be a kid.”

Laura Smith, who is a supporter of our Fast Forward campaign, is also pressing for fast access to Translarna for her son, Callum.

“When Callum was diagnosed with Duchenne, there were no treatments that could effectively slow the progression of his condition. But with Translarna, we are now within touching distance.

“When we heard NHS England had put their assessment of Translarna on hold, we were deeply upset. Whilst the NHS can afford to wait, boys like Callum cannot. We need urgent action to fast track the introduction of Translarna”.

Harry Barnley could also benefit from Translarna. Harry’s mum, Sue, said:

If you spend any time with Harry he will make you smile: he’s the cheeky chappy. He currently plays football after school, goes swimming and takes great delight in winding up his 7 year old brother, Jack.

Harry is one of the boys with Duchenne who could benefit from Translarna. It was devastating to hear out of the blue this week that NHS England has put its decision on the drug on hold, at a time when countries including Italy and Spain are now making it available. The NHS must make sure Translarna can reach boys like Harry. There’s no time for delay.

Get involved

You can join the fight for access to Translarna by:

If anyone is feeling anxious about this decision, we are here to support you. Please call our helpline on 020 7803 4800. The helpline is manned from 8.30am to 6.00pm from Monday to Friday.

 

If you have any queries regarding Translarna, please see our frequently asked questions.

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