Delays that left a six-year-old Leeds boy with only “goodwill” to pay for his treatment has had his case put before ministers.
Sam Brown is one of just 88 people in the UK with the rare Morquio syndrome, in which child growth slows and mobility is drastically reduced. While there is no cure to the life limiting condition, trials have shown the Vimizim drug can slow down the disease.
That hope though was taken from the Otley boy when the NHS dropped funding rather than face a legal challenge over how it hands out the cash, and now his family is left relying purely on the goodwill of the drug company, BioMarin.
Leeds MP Greg Mulholland, has told health ministers the families of this and other ultra-rare diseases cannot wait for another NHS drugs funding strategy.
He said: “There is a moral and a potentially legal responsibility to have these decisions made, we are already a month past the date at which these families were told there will be a decision, a potentially life changing decisions. Any delay in guaranteeing drug access could cause deterioration of their conditions, and this would be irreversible.”
Sam’s mother Katy said the NHS was failing to set out just how it will target ultra-rare diseases.
She said: “Children like Sam have been thrown into a distressing and unnecessary limbo. An innocent six year-old boy should not be suffering the consequences of this unacceptable delay, but sadly he and other Morquio sufferers are.”
Health minister George Freeman the NHS had to “operate with finite resources” and that any funding for one condition would come at the cost to another.
But he added: “We are determined that the case of these children is heard.”
RARE DISEASE LIMITS GROWTH
Morquio syndrome effects just a handful of children, but its effects can be devastating for the families concerned.
The inherited disease starts to be seen as the child grows up, with radically reduced growth and eventually severely reduced mobility.
The life limiting condition is caused by a failure of the body to breakdown certain sugars, which then build up in the body and damage organs.
There is no cure for the condition.