A SCHOOLBOY who suffers from a life-shortening disease will head to 10 Downing Street today to hand over petition for potentially life-changing drugs to be made available on the NHS.

Isaac White, 11, was diagnosed with the rare condition Duchenne Muscular Dystrophy when he was four years old.

But the drug Translarna could stop the disease from progressing, and massively improve Isaac’s life expectancy and quality of life, his parents believe.

The petition, appealing for the drug to be made available on the NHS, has collected 22,000 signatures.

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Issac, from Marston, Oxford, a pupil at John Watson School in Wheatley, said: “This drug is going to make a big difference and I’m excited about going to Downing Street.”

Duchenne Muscular Dystrophy mainly affects boys and results in rapid muscle degeneration and eventual death.

Since being diagnosed with Duchenne in 2008 Isaac has had issues walking long distances, and often requires the use of a motorised chair or walking aids when travelling longer distances.

There are about 2,400 boys in the UK with the condition and the average life expectancy of a sufferer is 25.

But it is hoped that Translarna, which is still going through trials, could stop the progression of the disease in boys like Isaac.

Isaac’s mother Kathy Wedell, 50, said: “Isaac has been on a clinical trial for the drug at Great Ormond Street Hospital for a year.

“We don’t know whether he has received the drug as he might have been given a placebo, but since he’s been on the trial his condition has not worsened when before he was deteriorating rapidly.”

The European Medical Agency approved the use of the drug in July 2014, and since then Germany and Italy have made the drug available on their national health services.

Mrs Wedell hopes that the UK Government will approve the drug, which will cost about £150,000 a year per patient, later this month.

Her husband, Stuart White, 48, Associate Professor in Politics at Oxford University, said: “In a word, Translarna is hope.

“Up to now there has been no treatment to tackle the underlying genetic condition and slow the muscle-wasting which Duchenne causes.

“Translarna offers hope of a longer and much better quality of life for Isaac and many others with Duchenne.”

Charity Action Duchenne’s chief executive Paul Lenihan said: “This new medicine was given EU marketing approval last July.

“However with NHS England’s funding approval process now stalled, there is a significant risk that this treatment will not be made available this year to those young men such as Isaac.

“Without this treatment, the disease will continue unabated.”

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