We cannot imagine our happy, smiley, Dua lose that beautiful smile.

Our beautiful daughter Dua has been diagnosed with a very rare degenerative genetic disorder known as SPG50 and doesn’t walk or talk. Dua is one of two children in the UK with SPG50. Quadriplegic by the age of 20 and wheelchair dependent by the age of 10 are words no parents wants to hear. We want to continue to see progression, however unfortunately this cruel disease will cause paralysis in her limbs . If Dua gets on the trial for gene therapy we are hoping to see a halt to her symptoms worsening and instead we will continue to see progression. It's hard to see your child deteriorate.

Today there is not treatment or drug to help these children. Dua has one protein missing - just one - but enough to cause such dire consequences.

Understandably, the diagnosis has shattered us , we always knew there was something but we never imagined it would an awful disease such as this one where our daughter will lose all her skills. We cannot imagine our happy, smiley Dua losing that beautiful smile of hers or not recognizing us.

Whilst there is no cure at present, a superhero Terry Pirovolakis has made it his mission to find a cure and clinical trials have begun in USA for gene therapy. His son has had the first dose.The FDA have approved a clinical trial for this treatment and Dua has been registered and 5 other children have had it with positive results. The cost of this is huge, however, and something that we will not be able to afford without everyones help . We need to raise this money so hopefully Dua will be selected. Dua is a perfect candidate.

We really need everyones help on gathering these funds so that our daughter is given a chance to be cured and in the meantime to give her the best chance in retaining movement in her body. The cost of the clinical trial is in the millions in fact and we are determined for Dua and all the other affected children to be cured. If the trials are successful then the therapy will become FDA approved and will be available to all at a cost.

Please help us save our daughter as we cannot imagine the alternative.