Muscular dystrophy boy in PM appeal


Published: Tuesday 13th January 2015 by The News Editor

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A nine year-old boy who has muscular dystrophy is handing in his personal plea to Prime Minister David Cameron to help him.

Archie Hill, of Gerrards Cross, Buckinghamshire, now has 22,785 signatures on the petition which he and families affected by the muscle-wasting condition helped to create. It calls for the NHS to end delays to a breakthrough treatment which will allow him to keep walking for longer.

Archie, who was diagnosed with the severe and progressive Duchenne muscular dystrophy at the age of three, is handing in the petition and his personal letter to the Prime Minister at 10 Downing Street tomorrow.

His condition causes irreversible muscle damage and leaves many boys needing a wheelchair by the time they are eight or nine years old. Problems with heart and lung muscles mean that few with the condition currently live to see the age of 30.

Archie is among a number of children who could benefit from Translarna, the first ever drug tackling the causes of Duchenne muscular dystrophy.

The drug was approved in Europe in August. It is currently available to boys in France, Spain, Germany, Italy and Denmark, but UK families – backed by the Muscular Dystrophy Campaign – claim they are being left to suffer agonising delays dues to NHS administrative issues.

Campaigners point out that to be eligible for the treatment, Translarna, these boys must still be able to walk. Each day this treatment is delayed results in a greater risk that they could miss out on the drug entirely.

Archie’s mother Louisa recalled that her family’s world “fell apart” when he was diagnosed and that “this delay is disastrous for Archie and others like him”.

She said: “Their muscles are constantly deteriorating and treatment is limited only to those who can still walk, so every day counts. Our boy will miss out on the opportunity to receive treatment; simply because of a delay caused by a review of business processes; which seems ludicrously unjust.

“In the spirit of remaining committed to doing something productive to help Archie, we are taking our plea to Downing Street and we thank all 22,785 people who have signed the petition so far.

“Time is of the essence for Archie, and accessing treatment quickly will give him extra precious time doing what he loves most; playing with his friends and joining them on the pitch playing football for Chalfont St Peter Under 9s.”

Muscular Dystrophy Campaign chief executive Robert Meadowcroft described Archie’s efforts in writing to the the Prime Minister to share his story as “really impressive”.

He said: “Translarna is a long-awaited breakthrough. We must see an end to the delays in this drug reaching children like Archie who desperately need it.”

Families who have been affected by Duchenne muscular dystrophy are also set to meet with MPs and peers in Westminster before helping to hand over the petition.

Translarna has been designed for 10-15% of the 2,500 children and adults in the UK who have Duchenne muscular dystrophy caused by a “nonsense” or ” stop” mutation.

Translarna will only be able to be used in boys over five years of age and who can still walk and only in cases caused by nonsense mutations – when a single letter of the DNA is changed which places a “stop signal” in the middle of the gene.

The drug can encourage cells to ignore this stop signal and read all the genetic information. In boys with Duchenne muscular dystrophy, this could restore dystrophin protein production in the muscles.

Published: Tuesday 13th January 2015 by The News Editor

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