The family of a 10-year-old boy with a life-limiting condition have told ITV News they are "devastated" after the health watchdog refused to grant access to a breakthrough drug to NHS patients in England.
Schoolboy Archie Hill was three when he was diagnosed with Duchenne Muscular Dystrophy - a muscle-weakening condition that has an average life expectancy of 27, and could see him needing a wheelchair in just a few years.
Video report by ITV News reporter Rebecca Barry:
His family have appealed for Translarna, a drug that slows the progress of the condition and has already been approved and funded in Scotland as well as a host of other European countries, to be made available on the NHS.
Their high-profile campaign has seen them gain the support of thousands, and led to the family taking their campaign directly to the Prime Minister with 50 other young sufferers.
But hopes for the drug - which can cost up to £400,000 per patient a year -to be rolled out UK-wide have been dashed by the National Institute for Health and Care Excellence (NICE), who have asked for further justification of the benefits and price of it.
NICE said it was "disappointed not to be able to recommend ataluren [also known as Translarna] " but was "not convinced" the drug was that beneficial to patients compared to standard treatments.
The body recognised a "clear need for a treatment that can prolong a child’s ability to walk but without serious side-effects".
NICE issued its draft guidance after considering "the evidence (and) opinions of the clinical and patient experts", with a final decision due to be made at a meeting on November 17.
After the decision, Archie told ITV News' Rebecca Barry he intends to keep fighting for access to the drug "so I can keep playing football with my friends".
Earlier, his mother Louisa said her son was "very upset" at the decision, and blamed authorities for "prioritising cost" over children's welfare.
Earlier this week, David Cameron was asked about the drug by Archie's local MP, Cheryl Gillan, in Parliament.
He said the decision should be left up to "clinicians rather than politicians" and called on the drug industry to find a way to lower the cost.
Archie's family say they will keep fighting to have Translarna made available in England, and believe change is needed urgently due to the risk that his condition could deteriorate rapidly.
What is Duchenne Muscular Dystrophy?
DMD a genetic condition which affects muscles, causing weakness
It typically affects boys, with 1 in 3,500 being diagnosed every year
Gradually the muscles get weaker so walking can be affected and chest and breathing problems can also develop as they get older
At present, the average life expectancy for those with DMD is 27
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said:"It is a bitter blow for all of us.
"Having spoken to some of the families affected, it really is impossible to describe their heartbreak, particularly as just last week Lothian NHS Trust approved the drug for a family in Scotland.
"Many parents across the rest of the UK are watching their child weaken and lose mobility day by day, as this really is a race against time.
"If we are not careful, it will be too late for many of these boys to even be eligible to take Translarna."