Do Something Good Today – Morquio Syndrome

29 Jan

Tia2

This little beauty is Tia (no, not the pony!)

Kamal.1

This is Kamal – how could anyone resist that grin?

959236612.1

And here is Sam, what a cutie.

They all have something in common, they have Morquio Syndrome.

They have what? I hear you say. Well, that’s what I said too, but then I got to know the mum of one of these gorgeous children and the story behind these smiles is a frightening one.

Basically, Morquio is what is termed an Ultra Rare Condition (also known as an Ultra Orphan Disease) and there are fewer than 120 sufferers in the whole of the UK. A person with Morquio starts to show symptoms usually around age 2-3 when their skeleton doesn’t grow at the same rate as their internal organs and problems start. I’m no medical expert so please excuse me for copying some relevant facts about the sort of things people with this condition have to deal with:-

Skeletal deformities
Breathing problems
Short trunk
Heart valve problems
Weakness in the neck
Muscle weakness
Spinal cord compression
Eye problems
Knock knees
Hearing loss
Overly flexible joints
Dental problems
Abnormal walk
Enlarged liver
Spinal problems
Enlarged spleen
Chest deformities
Limited energy
Or, as this paragraph says it:-
Morquio A (also called MPS IVA) is an inherited condition that affects major organ systems in the body. People born with Morquio A are missing or don’t make enough of an important enzyme and, as a result, they can’t break down certain materials in the body. These materials eventually build up in tissues, bones, and major organ systems. Morquio A can cause potentially serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, difficulty breathing, and early death.
Not much to smile about eh? Well, you can see by the photos above that having Morquio does not stop these young people from enjoying themselves. They are just like any other children, they have school, friends, homework, and out of school activities like swimming, drama and football. What they also have is exhaustion, pain, and frustration, and that’s just the tip of the iceberg.
Well, into the mix comes a wonder drug called Vimizim. A weekly infusion of this can – and does – seriously improve their quality of life. Using Vimizim means that they are less tired, less dependent on their wheelchairs, and overall in better shape. It helps them to breathe more easily, and there is a very real possibility that it will prolong life. It’s not some placebo that has been shown not to work, it makes a measurable and encouraging difference to life.
Now, if these children had, say, cystic fibrosis, which unfortunately is quite common, their treatment would be funded. I know with CF that a lot of the physiotherapy can and is done at home, but I also know there are way too many hospital and clinic appointments involved in ensuring that a person with CF gets the best possible chance at a good life, and the treatments have been shown to work, with the life span of CF sufferers rising all the time.
So if I told you that people with Morquio are being told that a life-enhancing and in all likelihood life-lengthening drug is seriously under threat of being withdrawn, what message would that send? Personally I feel that it’s telling people with Morquio (and bear in mind some of these are adults who have independent lives and contribute to our economy by holding down good jobs) that their lives are somehow worth less than the lives of others. How can that possibly be right? Or morally acceptable? These people aren’t asking for fancier wheels on their wheelchairs or anything that could possibly be seen as an “extra”, they just want the chance to live the best and fullest lives they can, in as little pain as possible, and, in the case of the children pictured above and others like them, to have the chance to grow up into the adults they deserve to be.
The NHS has not yet even bothered responding to the drug company, and obviously it is vital that they do so in order to ensure the continued supply of this essential medication.
So I’m asking you to help. To help Tia, and Kamal, and Sam, and all the other 120 people in the UK that live with this condition, and for the time it takes for your kettle to boil, or probably even less, please read and sign this petition.
The more people that have signed, the greater the weight this petition will have, it’s that simple.
If you have Facebook then please use the link on the petition to share it and ask your friends to sign. Please share it on Twitter as well, we need the numbers to make a difference. I’ve heard it said that the way to judge a nation is by how it treats the most vulnerable in its society. Let us show the powers that be that we judge all humans to be born equal and to have the right to what their country’s health care can provide, without prejudice.
Thanks for reading.
Information about Morquio taken from http://www.vimizim.com
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4 Responses to “Do Something Good Today – Morquio Syndrome”

  1. mummyshambles January 29, 2015 at 4:22 pm #

    Signed and shared my lovely. xXx

  2. mamamrsme February 4, 2015 at 8:50 am #

    Signed and shared xx

    • ouremuk66 February 4, 2015 at 9:30 am #

      Thank you, much appreciated xx

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