The mum of Otley’s six-year-old Sam Brown - who has the rare and incurable Morquio Syndrome - has penned a heartfelt letter urging health chiefs to bring forward their decision on approving a pioneering drug.
As reported by the YEP last week, Morquio campaigners were finally given hope after health regulator NICE said they were now considering approving Vimizim - which patients on clinical trials claim has transformed their lives.
But NICE also admitted that the final decision would now be delayed from October to January. Katy Brown has written to the Prime Minister and chief executive and deputy chief executive of NICE urging them to review those timescales. She said, while she was “very relieved” to learn they are now considering funding Vimizim, there was “significant disappointment” with the extra delay.
She wrote: “This may seem trivial to you, but for families it is a cruel twist. It prolongs the torture of limbo which is emotionally debilitating, and also physically damaging for those sufferers not on the trial who remain untreated and without access to the drug.”
Katy urged decision-makers to consider the well-being of patients and their families who “have endured an horrendous year on a painful and often unnecessary bureaucratic roller coaster - and bring this matter to conclusion as swiftly as is humanly possible”.