What Vimizim means to Lily-May

What does Vimizim mean to Lily-May who had a late diagnoses just a couple of weeks after the trial closed? Lily-May hasn’t had the opportunity to receive treatment yet – even though Vimizim was licensed in April 2014. Read her compelling letter to David Cameron to understand what Vimizim means to her.

Dear Mr David Cameron,

I am writing to you regarding the funding for the medication called ‘Vimizim’.

I am a thirteen year old girl, who has had Morquio for all of my life. I however I have had several misdiagnoses, and I was only diagnosed with Morquio 4a three years ago. It has however affected my life greatly.

When I was younger, if we went shopping I would be complaining of leg ache, and tiredness. Being merely a child, we all believed it was because I was a young child not wanting to go shopping. We used to go out into town to do market shopping every Saturday. I now miss doing this, and cannot remember the last time that I did indeed go around town walking.

Whenever I go into town now in my wheelchair, or even out for a family meal, I get people staring at me as if I am some kind of rotten apple on the pavement. People pull on the back of my chair, and start to shake it. Sometimes if I do go into the supermarket (still in my power chair) people will purposefully knock their trolley into the back of my chair. I need to use a chair because I cannot walk more than a few metres without excruciating pain and extreme tiredness that will lead to me having to have several days in bed to recover my strength. I cannot go shopping or to the cinema with my friends without a great deal of assistance and support.

Morquio has not just affected me, but my whole family. I may be the only one going through the physical pain, but my family suffer just as much. Without my mum, dad and little brother, I truly do not know where I would be now. They have helped me with everything that I remember.

It is with due regret that I am writing to you today. I know that this will go through your Secretary first, and will not be at the top of your to-do pile. However I felt it was worth a try. I understand that the elections are coming up soon, and that this may feel like an inconvenience to you. Not just me, but others like me, in the same position, we have been put through this constant torment of non stop pain, no cure, waiting, and still waiting. We were promised an answer to whether the funding for Vimizin would go ahead in December. Like many before me have said, we were left in ‘limbo’. Only to be told that we would have to wait until April time this year. Shortly after this, we were told that we would then have to wait until after the elections. Someone has said that we may have to wait until June time.

I have not been diagnosed for as long as others, nor is my condition as serious as some. Yet this does not stop me from being me does it? I have been so limited to a wheelchair, aids around the house, rules such as ‘you can’t go roller-skating or ice-skating in case you fall.’ that I do not truly know what ‘normal’ is like.

Normal is a big subject. This is because everyone has a different opinion of normal. Mine is someone who has the ability to run, walk, jump, hop, skip etc. Someone who can stand on their own two legs and not need any support. Someone who can confidently say that they can do P.E. I can’t run. Walk. Jump. Hop. Skip. Or stand on my own two legs without support. Neither can I do P.E.

Moving around my own home is often a struggle. Even to just walk to my room, I hold onto shelves and press against walls. We have had a stair lift installed, bathroom adjusted, a chair moved into the kitchen so that I can sit more comfortable when eating, a chair in the living room so that I can be more comfortable. I even have a profiling bed in my bedroom. I cannot lay flat, otherwise my airways close up and I can’t breathe. It also helps to relieve the pain that I have in my hips.

I currently am at Sheffield Children’s Hospital and Manchester Children’s Hospital. I am soon to be going to Wigan to see a specialist hip surgeon. My hips are migrating and I need to have some form of hip replacement.

There is another symptom that Morquio causes. Cloudy corneas. This is the deterioration of a part of the eye. This means that I am susceptible to bright lights. This also means that my eyes, when I look through them, appear to be misted. As I get older, my eyes will become more clouded, causing me to possibly eventually go blind.

All of the above symptoms that I have mentioned are only the tip of the iceberg. There is more going on inside me, causing me more pain. However Vimizim can help reduce and slow down these symptoms. Therefore reducing my pain, increasing my mobility, and extending my life.

Unfortunately, I was diagnosed too late to participate in the trials. I have however heard of the relief and freedom that it has given to others, such as Sam Brown and Archie, Isaac. And Gracie.

I could have had compassionate access to the drug for a period of 5 years but NHS England have ignored the offer made by the wonderful people who make Vimizim. Are they saying that my life is worth nothing? Or are they scared that the drug will make such a difference to our lives that they will have no choice other than to fund it? Are they as frightened as me though I wonder…

The only part of my body not affected by Morquio, is my brain. I am fully aware of what is going on in my body, and the long term damage that is constantly being done. I am a high-acheiving grammar school student with ambitions to become a scientist. But without the help of a drug like Vimizim, I will have to struggle even harder. Since mid November, I have had more time off school than in school due to the amount of pain that I am constantly in. Despite this, I am keeping up with my studies, due to the support of my school, as I am working from home. My point being I have a brain, I know how to use it, but I need YOUR help to give me the drug I need to be able to keep doing this because sometimes I am so tired that to be brutally honest, giving up would definitely be the easiest option.

We will all keep fighting for Vimizim no matter how far it goes. None of us want to live like this for longer than we have to. All of us deserve a chance. All of us deserve a feeling of normality. I am a fighter, because I have no choice and I have everything to fight for. Along with my family I have been fighting for the last few years and I will not stop now, just because you don’t think I’m worth it.

Please reply to my family, mine and all of my fellow sufferers.

We all trust the quality of our lives into your hands, hoping that you will do the right thing.

Yours Sincerely,

Lily-May Clayton