Drugs fight goes on for Leeds boy as NHS delays cash

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 Leeds 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”.

But he added: “We are determined that the case of these children is heard.”