The mother of a boy with an ultra-rare condition said today she was “relieved and delighted” after health chiefs agreed to fund drugs to treat him and other patients on the NHS.
Campaigners have been pushing for Vimizim, used to treat those with the ultra-rare disease Morquio Syndrome, to be available on the NHS and today health regulator NICE published final guidance saying the drug is now officially approved for use in the UK and can be funded by NHS England.
Katy Brown, whose son Sam, seven, of Otley, has been treated with Vimizim free, thanks to the goodwill of drug company BioMarin, welcomed the announcement.
“We are relieved and delighted by today’s decision,” she said.
“We are now looking forward to a relaxing family Christmas, celebrating this victory and a much brighter future for Sam.”
Morquio Syndrome is a degenerative condition which limits growth and mobility and shortens life expectancy to an average of 25 years.
Sam is one of just 88 people in England with Morquio Syndrome and the bid to get the drug funded on the NHS has been backed by Leeds North West MP, Greg Mulholland.
“This is exactly the news we wanted and I am delighted for Sam and for all the people with Morquio who will now get access to the treatment they need,” Mr Mulholland said.
Patients on clinical trails have claimed Vimizim has transformed their lives.