One of the country’s oldest sufferers of the rare Morquio Syndrome has welcomed a temporary extension in access to a life-changing drug.
But Angela Paton, 41, of Kippax, said she and others with the syndrome - just 105 people in the UK - are “not out of the woods” until NHS England agree to longer-term funding of Vimizim.
Those on the original clinical trial have been provided with Vimizim free of charge in a goodwill gesture by drug company BioMarin, while NHS bosses stalled over making a decision on its future funding.
Last month BioMarin said that, due to the NHS delays, it would be forced to end that goodwill on May 12, but that deadline has now been extended to June 25 - when it is understood NHS England are set to make a decision.
Angela said they have been thrown a “temporary lifeline”.
She said: “My first reaction was just a huge sigh of relief. We were within just two weeks of losing Vimizim so it ‘s fantastic we now have eight weeks left of treatment and more importantly we finally have a definitive answer as to when NHS England will make their decision
“However, this does not, by any means, mean we are out of the woods. This still doesn’t provide Vimizim for the many adults and children that were not on the clinical trial. That will only come if NHS England make a positive decision on funding on June 25.”
The inherited condition limits growth and mobility and shortens life expectancy to an average age of 25. While there is no cure, trials have shown Vimizim can slow down the disease. On Friday, Katy Brown, mum of six-year-old Sam Brown, of Otley, who also has the condition, raised the issue with David Cameron while he was up in Leeds.
Speaking afterwards, she said: “Let’s hope this is now the first of many more positive steps for Sam, and all Morquio sufferers in the fight for Vimizim.”