Fund­ing would help suf­fer­ers of mus­cu­lar dis­tro­phy

Buckinghamshire Advertiser - - FRONT PAGE - by Tom Her­bert tom.her­bert@trin­i­tymir­ror.com Twit­ter: @TRHer­bert

A FAM­ILY from Ger­rards Cross face an anx­ious wait for a de­ci­sion that could help im­prove their lives.

Louisa and Gary Hill, par­ents to 10-yearold mus­cu­lar dys­tro­phy suf­ferer Archie, spoke of their fears ahead of a lifechang­ing meet­ing on Tues­day night.

The fam­ily fear a de­ci­sion to fund the drug Translarna – which would en­hance Archie’s con­di­tion – would be re­jected by gov­ern­ment body NICE.

Translarna is said to be the first drug to tar­get the cause of this life-lim­it­ing con­di­tion, and is said to keep boys like Archie walk­ing for longer.

NICE will meet to re­view whether the drug should be funded on the NHS. The Hills will hear be­fore Christ­mas about NICE’s de­ci­sion and whether the drug will be made avail­able for Archie and 50 other boys like him.

Last month the fam­ily were left dev­as­tated af­ter be­ing told Translarna would not be rec­om­mended on the NHS with­out clar­i­fi­ca­tion of the costs in­volved.

Mr Hill said: “Our great­est fear is that NICE will de­cide that Translarna is too ex­pen­sive, and Archie and the other boys will miss out. This drug could give them longer to do the things that are in­cred­i­bly im­por­tant for a child.

“All Archie wants to do is play foot­ball and keep up with his friends. It’s not like there are lots of other ther­a­pies out there for Duchenne.

“At the mo­ment steroids and phys­io­ther­apy are about all there is to keep chil­dren mo­bile. It’s been such a tough wait for all the fam­i­lies in­volved.”

The drug has been made avail­able in coun­tries in­clud­ing Ger­many, France, Spain and Italy, but fam­i­lies across the UK have faced waits for news on fund­ing.

Robert Mead­owcroft, chief ex­ec­u­tive of Mus­cu­lar Dys­tro­phy UK, said: “When NICE meets, we urge them to do all within their power to find a work­able so­lu­tion for de­liv­er­ing on this long-awaited break­through. Archie’s child­hood, and those of many other el­i­gi­ble young peo­ple across the UK, could be trans­formed.

“It is deeply en­cour­ag­ing that to­day, more and more ther­a­pies for Duchenne mus­cu­lar dys­tro­phy are reach­ing clin­i­cal tri­als. As Duchenne is pro­gres­sive, we can­not af­ford to go through this con­vo­luted and painful process each and ev­ery time a new ther­apy is in sight. The emo­tional cost for fam­i­lies sim­ply can­not be jus­ti­fied.”

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