Sam’s race against time for drug

Sam Brown, six, with  parents Katy and Simon from Otley. Sam has Morquio syndrome. Picture by Tony Johnson
Sam Brown, six, with parents Katy and Simon from Otley. Sam has Morquio syndrome. Picture by Tony Johnson
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A mother has issued a heartfelt plea to NHS chiefs to fund a new treatment for an incurable degenerative condition which is her son’s only hope of a better life.

Sam Brown, six, of Otley, was born with Morquio syndrome, which prevents children growing normally with a maximum height of 3.5ft. Most need wheelchairs by their teens and do not survive beyond their 20s.

Sam Brown from Otley has Morquio syndrome.

Sam Brown from Otley has Morquio syndrome.

Since 2012, his family have been given fresh hope in a trial of a new drug Vimizim. They say it has led to significant improvements in his condition, which affects fewer than 100 people in the UK. The youngster is continuing to receive the treatment ahead of a meeting of health officials next month to decide if it should be funded by taxpayers.

But his family have been devastated to learn funding for the drug is unlikely to be approved.

The family’s MP Greg Mulholland has secured a meeting with Life Sciences Minister George Freeman to discuss his plight as a campaign for funding steps up.

His mother Katy, 38, said: “It has benefited him hugely. It’s not a cure. It will certainly extend his quality of life but, based on what I’ve seen, there’s a good chance it will extend his life and allow him to be independent.”

But she added: “Every indication we have been given thus far is that the funding will not be approved, which means his treatment would stop and the disease would be allowed to progress. This is unthinkable.

“Sam has a very limited timeframe for the right decision to be made. He has no idea of the silent path Morquio syndrome has set for his future. With Vimizim, he may never need to know.”

A regional spokesman for NHS England said decisions to fund treatments were made on their effectiveness following trials. An announcement would be made next month.

A petition calling on NHS England to fund the drug is at http://you.38degrees.org.uk/petitions/nhs-england-s-scorecard-discriminates-against-treating-ultra-rare-diseases.

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