Fam­ily cel­e­brates ‘best present’ for all boys who need treat­ment

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

A DEAL has been struck be­tween NHS England and phar­ma­ceu­ti­cal firm PTC Ther­a­peu­tics, al­low­ing chil­dren to re­ceive a break­through mus­cu­lar drug within months.

Just over 50 boys in England are now el­i­gi­ble to re­ceive Translarna – a drug which could keep those with the con­di­tion walk­ing longer – thanks to the ground­break­ing deal.

Fam­i­lies across England have faced an ag­o­nis­ing two year wait for an NHS de­ci­sion and now the process be­gins of de­liv­er­ing the treat­ment to those who need it.

In April NICE gave NHS England and phar­ma­ceu­ti­cal firm, PTC Ther­a­peu­tics, un­til Thurs­day July 7 to agree a deal on Translarna, and they are now ex­pected to for­mally pub­lish guid­ance within weeks.

Chal­font St Peter based Louisa Hill, whose son Archie has the con­di­tion, said: “It’s ab­so­lutely fan­tas­tic news, it’s the best I could have asked for.

“I rang and told Archie and his re­ac­tion was ‘wow’ – he’s ab­so­lutely thrilled to bits.

“We’re de­lighted about the de­ci­sion and can’t ac­tu­ally be­lieve the boys will be re­ceiv­ing it. It’s a shame it’s taken so long but it will help them so much.”

Mrs Hill said she wants to give all of the chil­dren that are el­i­gi­ble ‘a big hug be­cause I feel so proud that we have made a dif­fer­ence’ and thanks go to NICE, NHS England, lo­cal MPs and Mus­cu­lar Dys­tro­phy UK.

She added: “Archie has been strug­gling men­tally over the last few weeks.

“He doesn’t like be­ing dif­fer­ent – he said he’s scared he will wake up one day and he can’t walk.”

Mrs Hill cel­e­brated her birth­day on Fri­day (July 8) and said it is the ‘best present I could asked for’.

Robert Mead­owcroft, chief ex­ec­u­tive of Mus­cu­lar Dys­tro­phy UK, said: “We are de­lighted with this long-awaited news that an agree­ment has fi­nally been reached be­tween NHS England and PTC Ther­a­peu­tics.

“While NICE and NHS England have been con­duct­ing their as­sess­ment of Translarna, fam­i­lies have faced a des­per­ately long and frus­trat­ing wait, and had to en­dure the heart­break of see­ing their sons de­cline, while this li­censed drug was not avail­able to them.

“We give the boys and their fam­i­lies our huge thanks and recog­ni­tion of their per­sis­tent and ded­i­cated cam­paign­ing ef­forts and, af­ter work­ing to­gether with many char­i­ties, we are pleased we are now a sig­nif­i­cant step closer to Translarna at last be­ing made avail­able to el­i­gi­ble boys on the NHS in England.

“How­ever, the fight goes on for equal­ity of ac­cess to el­i­gi­ble boys in all coun­tries of the UK not least in Scot­land where fam­i­lies and Mus­cu­lar Dys­tro­phy UK have to­gether urged the First Min­is­ter to act de­ci­sively and ur­gently to end the cur­rent ‘post­code lot­tery’.

“In Wales and North­ern Ire­land, there is a clear un­der­stand­ing they will fol­low NICE guid­ance when it is pub­lished.”

Cam­paign: Archie Hill with his mum Louisa and dad Gary. Be­low, Archie takes his fight to the top with a visit to Down­ing Street

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