Boy hoping NHS will fund drugs
HealthH chiefs’ life-changing decision
A FAMILY is waiting on a li life-changing decision fr from NHS England chiefs who are to decide whether or not to fund a b breakthrough drug.
As the Advertiser went to press, Archie Hill, from G Gerrards Cross, who suffers fr from rare muscle wasting co condition Duchenne m muscular dystrophy, is waiting to hear news about new drug Translarna.
Translarna, which became the first drug to address the underlying cause of Duchenne muscular dystrophy, was approved for use in the EU in August last year.
It has been available in several European countries since last year, but parents in the UK have had to wait to hear the news if NHS England will fund it.
Archie’s mum Louisa said: “It would just mean so much for all those boys.
“I’m not going to give up. I’m going to keep going.
“I just want some answers, and what you can do to get it licensed here.”
Mrs Hill is ‘50/50’ about whether she thinks the drug will get licensed.
She said: “It’s just so important. There’s a drug out there that will allow Archie to be a child for a little bit longer. If it’s a no I will keep fighting and I really won’t give up. We will get there. I’m on a mission.”
Peter Sutton, policy and campaigns officer at Muscular Dystrophy UK, said: “Currently, the only treatment usually given to children is steroids. However, these are accompanied by severe side effects, including mood swings, weight gain and thinning bones.
“Clinical trials of Translarna indicate it would keep children walking for longer and delay the need for a wheelchair. However, in order to access the drug these children must still be walking. Time is so tight – every day of delay means children come closer to losing the ability to walk and thus ineligible for Translarna.”
CheshamandAmersham MP Cheryl Gillan said: “For families like the Hills, every moment is precious and I hope that the funding review will be completed with the utmost speed.”
Mrs Hill added: “I’m going to do all I can. I’m going to keep on fighting. I just do it for Archie and I want to make a difference for all the boys.”