This is our 18 month old little girl Wynter, born “perfectly healthy” so we thought, but our world came crashing down when at 3.5 months old, she was diagnosed with a terminal illness called Spinal Muscular Atrophy (SMA) type 1. This disease is a rare infantile version of motor neurone disease that results in motor neurone loss and muscle wasting, eventually leading to death. This disease affects every muscle in the body, making it difficult to eat and breathe. Our little girl is unable to sit, crawl, walk, run or eat properly because of this disease, and it is heartbreaking for us to watch her struggles with what most of us take for granted. The normal life expectancy of infants with type 1 SMA is usually no more than 2 years. In saying this, we found hope in that there was now a treatment option available in Australia called Spinraza that came out last year that has given the potential for us to have her for longer than the historical life expectancy but it is still very unknown. Wynter has been receiving Spinraza since she was 4 months old - this drug is given via a lumber puncture and so far she has had 6 doses. Although we are incredibly grateful for this drug, and have seen some great improvements in her motor skills, it is difficult to continually watch her go through pain and the unknown of whether it will cause future problems health wise or whether it could be taken away from us in the blink of an eye. Until recently Spinraza was the only treatment available for SMA patients, however a new gene replacement therapy, Zolgensma, has been FDA approved in the USA in May this year for children under two years of age. This treatment has shown amazing results and is a one time dose only, replacing the missing gene entirely. This drug we feel is the best on the market for SMA and we are desperately wanting to get it for our little girl. Naturally we as parents want the best treatment we can get for her, but we need your help. Wynter is 18 months old, therefore time is not on our side. We know this is a HUGE HUGE ask for this amount, and would not do it unless we felt it was lifechanging. We have rarely asked for anything and taken sole responsibility of the costs associated with having this disease, however this new treatment is something we cannot get for her ourselves, at over $3 million (AUD) price tag, it is currently the world's most expensive drug. We don't know if we can raise the funds to help our little girl but we owe it to her to try. If we do not reach our goal, the donations will help us explore other options like relocating to the US to try and access treatment there as well as getting Wynter the equipment and therapy she needs. If we have excess funds we will donate to charity. Thankyou from the bottom of our hearts for reading this.
- Jason Daly
- Rosanna Tesoro
- Anne Bowen
Fundraising team: Fundraising team (2)