Spinal Muscular Atrophy (SMA) is a rare, genetic neuromuscular condition causing progressive muscle wasting (atrophy) and weakness leading to loss of movement. This may affect crawling and walking ability, arm, hand, head and neck movement, breathing and swallowing. There are different forms of SMA and a wide spectrum of how severely children and adults are affected.
We live in the UK and there is a treatment available called Spinraza, which increases survival motor neuron (SMN) protein production. We are extremely grateful to have this treatment available to us, as only a few years ago, babies with SMA did not have this hope and would likely not live to reach the age of 2.
Although we have this treatment option available, there is a new pioneering much more advanced treatment called Zolgensma. Zolgensma is a gene therapy medicine for treating SMA. It targets the genetic root cause of SMA with a one-time-only dose and replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene. It is currently only available in the USA and with a large price tag, £1.2m!
We are reaching out to our family, friends and the general public to help us raise funds to get our son this life saving treatment. This can be done by donating, sharing our story to everyone and anyone you know, setting up fundraising ideas or events - whatever you can do will be truly appreciated. We can’t do this without you and will be eternally grateful for any donations or support, large or small.
Zolgensma will give Edward the best chance to live a fulfilling life. From a Mum and Dad who love their son more than anything in life, we thank you from the bottom of our hearts.
Megan and John xxx
All money raised will go to help for Edward. If we raise insufficient funds or surplus funds, the funds will be used, if appropriate, to fund for our child's needs or be donated to other SMA charities or fundraisers. If in those circumstances we are unable to use all or part of the funds for the benefit of our child, then any funds that cannot be used will be transferred to be used for the general charitable purpose of SMA or similar.
- Anna Ali
- Roz Wells
Organiser and beneficiary
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