What Vimizim means to Gracie

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What Does Vimizim Mean to Gracie?

Penned by Gracie’s parents, the following passage explains exactly how important Vimizim is:

The Trial in Our Eyes
Our beautiful and amazing daughter Gracie was born, without our knowledge at the time, with Morquio Disease. She deals with pain and discomfort all day, and is beginning to notice how she is different to her peers. Despite all her struggles, Gracie is the most cheerful little girl you could ever meet, and when asked how she is today, will always reply “brilliant!” As her parents we can often feel powerless to help her in any way, or to try to take her pain away, so when we heard about a medical trial, back in the winter of 2011, which could help to improve Gracie ‘s life, we did what we could to ensure that Gracie was included in the trial. At the time, not much was known about the drug-now called Vimizim- but what we did know was that we couldn’t let Gracie miss out on getting the best chance in life possible.

Gracie started the drugs trial in January 2012, when she was 5 years old. We were made fully aware that there were 3 strands to the trial – some patients would get the drug infused every week, some every other week, and some patients would simply be infused with a placebo. We of course hoped beyond hope that Gracie would at least be receiving the drug every other week, and now looking back, we believe that she was on it every week, as she reacted to something from a very early stage. As Gracie’s parents, we felt a huge sense of responsibility, asking her at such a young age to be attached to a drip for hours every Monday, and cannulated before each infusion. But Gracie took it all in her stride, and seemed to understand that this was something very special and important to be a part of.

Two and a half years down the road, and Gracie is now receiving Vimizim weekly, at Manchester Children’s Hospital. She has had a port-a-cath fitted in her chest, as cannulating her was getting more and more difficult, and it was taking anything up to 8 attempts to get the cannula in. Although our weekly journey takes us anything up to 2 hours each way, the positives outweigh the negatives, as Gracie loves going to hospital and has a very close relationship to the nursing team and play specialist. We have seen improvements in Gracie’s distance-walking, and she is still growing- in fact she is now above average height for a Morquio patient. Moreover, before the trial, Gracie was beginning to have clouding of her corneas, a common problem with Morquio, and now there is no evidence of any clouding at all, for 2 years running. On its own, this would only have stayed the same, or more realistically, got worse, so we are certain that this is a benefit of the drug for Gracie, even though this is not a benefit that the experts are that aware of.

When we heard the news that Vimizim had been approved, we were over the moon – it was a real reason to celebrate, and we felt immensely proud that Gracie had played a part in this trial. Finally we had a reason to hope for Gracie’s future, her hopes and dreams could hopefully be fulfilled.

But things are not that easy. We are now clinging to the hope that Vimizim will be funded so that Gracie and the other patients with Morquio in the UK can have access to this life-changing drug for the rest of their lives. We can’t even bear to think about the fact that it may not be funded. It would feel like we had given Gracie the best chance, to have it snatched away from her – someone who will give so much to society as the wonderful, positive human being that she is. Even if England decide to fund Vimizim, we live in Wales, so we are now forced with another major decision: if Wales decide not to fund the drug, we will have to move house into England so that Gracie can still access it. We are desperate for Gracie to continue attending the same school, as her friends are so important to her – they simply see her  as Gracie and look past her disability. The school are also extremely supportive. There are only 5 people in Wales with Morquio, including Gracie, so it’s difficult to try and guess whether or not this drug will be seen as ‘value for money’.

Looking to the future, the ideal solution would be for Gracie to have infusions at home, then eventually at school. This way she wouldn’t have to miss a full day of school every week. But none of this will be possible if the drug is not funded. It makes me angry when I think about all the money spent on drug and alcohol abuse, stop smoking campaigns etc. Yes, Vimizim is an expensive drug, but our daughter’s life is more than worth it.