STRENGTH IN NUMBERS

Strength in Numbers dedicated to my late mother Kay

‘Stopping my lifesaving drug will be my end,’ says woman with rare illness

Sarah Long, oldest survivor of Morquio syndrome, pleads for NHS to provide expensive drug to treat condition

Every day Sarah Long becomes weaker. She cannot sleep for more than an hour at a time, loses concentration and struggles to speak. “I don’t have much longer,” she says with a remarkable lack of self-pity. At 44, she is by far the oldest person to have Morquio syndrome, an extremely rare degenerative impairment, caused by missing enzymes, that has stopped her from growing since the age of six.

Most people with the syndrome die in their teens from a heart attack or because their lungs fail. Only 88 people in England – and 160 worldwide – are known to have the syndrome and barely a handful have made it into their 30s. Long has earned her surname. But, then, since she lost her mother when she was a teenager, she has been nothing if not strong-willed. She puts her longevity down to “bloodymindedness, a strong heart, determination – that was something my mum taught me”.

Not only did she go on to defy every medical prediction and reach her 40s, she took a degree in sociology, then a master’s, and is now in the middle of studying for a PhD. It was the same determination that in 2012 made her choose to test a free trial of a drug called Vimizim. She had spent eight months laid low with pneumonia, a period in which she says she “didn’t function”, and felt going on the trial was worth the gamble.

Click on the link to read more

http://www.theguardian.com/society/2015/jul/25/morquio-syndrome-nhs-drug-funding-sarah-long

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Filed under: Disabilities, NHS, , ,

Heartbroken Sam, 6, could lose the drug that gave him his childhood back

LITTLE Sam Brown loves nothing more than playing with his younger brother and dreams of becoming a fireman.

Yet six-year-old Sam has a rare illness that means he will never physically grow up and it threatens to rob him of his future.

A pioneering drug has been helping to keep him mobile for the past three-and-a-half years and offers the only hope of prolonging his life. Yet his parents must wait until the end of this month to learn if the NHS will take over the £395,000-a-year funding of his treatment, which until now has been provided free of charge by a drug company as part of a trial.

His mother Katy, 38, said: “This drug has given him the freedom to be a child again. “It feels like a ticking time bomb. If he doesn’t have that drug, the clock will start ticking and the degenerative impact can start to happen. “Once the damage is done no amount of this drug at a later date is going to turn back the clock. “There is no other viable option. We could raise a few hundred thousand pounds as a one-off but to do it year in year out for the rest of his life is not feasible. Not allowing him access to a proven treatment discriminates against people with rare illnesses. I will never stop fighting for him until he gets what he deserves.”

Click on the link to read more

http://www.express.co.uk/news/uk/584296/Sam-Brown-treatment-NHS-illness-Vimizim

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Little Sam has become much better after receiving the special treatment

Filed under: NHS, , ,

A mother’s plea to the NHS

With the NHS having suspended its procedure for approving vital treatments, sufferers of rare diseases have been left in limbo. Katy Brown tells her family’s story.

Let me introduce you to Sam. He is our six year old son and for the past two months we have been fighting to keep him smiling. More precisely, we have been fighting for the drug that he had been trialling for almost three years to be funded by the NHS. You see Sam has something called an ultra rare disease. That means that there are less than 500 sufferers in the country. Actually there are just 78 sufferers of Morquio Syndrome in England. Just 78.

How a disease with 20,000 sufferers is funded, researched, prioritised and understood is very, very different to one where there are just 78 people affected. Let me paint you a picture of Morquio. An adult the same size as a three year old. Organs squashed into a very small space. Severe physical disability. Hearing problems, sight problems, heart problems, lung problems. Life expectancy on average of 25. No treatment.

Please click on the link to read more

http://www.yorkshirepost.co.uk/news/features/a-mother-s-plea-to-the-nhs-1-7063396

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19 November 2014……. Sam Brown, 6, with parents Katy and Simon from Otley. Sam has Morquio syndrome. He is being treated with drugs thanks to a trial in Manchester which have significantly improved his condition but cash for this will run out next month and the NHS is unlikely to pick up the tab.TJ100584h Picture by Tony Johnson

Filed under: Uncategorized, , , ,

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