Families affected by ultra-rare Morquio Syndrome have spoken of their relief after a drug to treat the condition was reinstated.
Otley’s Sam Brown and Angela Paton, from Kippax, were among 34 people on a clinical trial of BioMarin’s drug Vimizin but its long-term future remains in the balance.
Supply of the drug was discontinued around a month ago but will now resume until the National Institute of Health and Care Excellence (NICE) publishes its final guidance, on whether or not to approve the drug for UK use, on October 28.
Those hit by Morquio, a rare degenerative condition caused by a missing enzyme, are relieved but fear for the future.
Katy Brown, mother of Sam, six, said her family is “incredibly relieved” at the reprieve.
She said: “This news is a hugely encouraging step, but a positive funding decision from the NHS is the only viable long-term solution.”
Angela, 41, has little confidence in NICE. She said: “I hope they prove me wrong and realise how important it is. We’ve seen in just four weeks since the treatment was withdrawn, the impact on people.”
Just 88 people in the UK have Morquio, which can lead to skeletal deformities, heart disease and loss of sight or hearing. Many people with the condition die in their teens.
Leeds North West MP Greg Mulholland talked of short-term relief, while feeling for those not on the trial. He said: “BioMarin have done their part, and I would urge NICE and NHS England to approve a drug that clearly works.”
Christine Lavery, of support charity the MPS Society, said she is “concerned for those diagnosed since the trial closed”.
NICE said its committee will meet before publishing guidance later this year.