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Renewed hope for Otley youngster with rare disease

Renewed hope for Otley youngster with rare disease

Published at 6:09pm 23rd November 2015. (Updated at 6:25pm 23rd November 2015)

The family of a 7-year-old Otley boy with a rare disease are welcoming an agreement to provide vital treatment, saying it's "a step in the right direction".

Sam Brown is one of just 88 people in England who have Morquio Syndrome.

Until now, he's been unable to receive the drug Vimizim through the NHS, with manufacturer BioMarin supplying it on a trial basis.

However, NHS England has negotiated a deal which will give patients access to the drug for five years.

The National Institute for Health and Care Excellence (NICE) has previously said that whilst Vimizim does improve some aspects of quality of life, the benefits are uncertain and it's also expensive.

Dame Barbara Hakin, NHS England's National Director of Commissioning Operations, said: "This agreement will enable us to start treating people with this debilitating condition, assess whether this treatment provides real benefits in the long term and, assuming it does, then continue to provide it in a sustainable way.

"This drug is not a cure, but it can have benefits for patients, though long-term outcomes remain uncertain.

"NICE was right to highlight the very high list price, which would have a disproportionate impact on the availability of other treatments. It is good news for all our patients that we have been able to come to a more reasonable arrangement that works for the NHS more broadly."

Otley's MP, Greg Mulholland, has been leading the #FundOurDrugsNOW campaign and raised the issue in Parliament.

Commenting on NHS England's announcement, he said: "After 12 months of campaigning, today's news is a huge victory. It will mean that those with Morquio Syndrome will finally be able to get the Vimizim drug through NHS England. Today's news is what many Morquio sufferers and their families have waited a long time to hear.

"It is great news for Sam Brown, whose parents Katy and Simon have campaigned long and hard. The agreement announced today is the breakthrough we all wanted.

"When rubber-stamped on 16th December, the agreement will mean the Vimizim drug will be immediately available to everyone diagnosed with condition. We are all delighted at today's news, it is exactly what we have been demanding for the last 12 months. I could not be happier for Sam and his family."

What is Morquio Syndrome?

It's a severely debilitating, inherited disorder caused by the lack of a key enzyme which ordinarily breaks down large sugar molecules the body cannot otherwise process.

The accumulation of these molecules in the body's cells causes symptoms including joint and skeletal abnormalities, hearing and vision loss, heart valve disease, pain, fatigue and reduced life expectancy.

On average, around three children are born with the disorder each year and they are only expected to live for around 25 years.

Vimizim is currently the only treatment available and works by replacing the defective enzyme through regular infusions.

The drug has been shown to slow progression of the symptoms and allow some patients a longer and better quality of life.