02/06/2026
We dont normally post other things on here but this has to be shared, can anyone please donate to get kole his chance at treatment, even £1 will make a massive difference in his journey.
Hi, Im Kole.
I’m 22months old and on the 28th April after spending practically all of my short life in and out of hospital, hours upon hours at specialist appointments. I was diagnosed with Sanfilippo Syndrome (MPSlllA) more commonly known as Childhood Dementia. Sanfilippo syndrome is a rare, genetic, neurodegenerative disorder. Affecting around only 140 children in the UK, my family believe I am the youngest nationwide battling the disease. Sanfilippo Syndrome is referred to as Childhood Dementia due to the nature of the disease. It attacks the central nervous system, which includes the brain and spinal cord. Slowly stealing children of skills they have gained. Without treatment I will loose all of my abilities leading to premature death by my mid teens.
HOWEVER, the FDA over in America have until the 19th September this year to approve a Gene Therapy treatment (UX111) clinically proven to improve my quality of life and time with my family. Children like me that have received the treatment in clinical trials are still walking, reading & playing amongst friends in sports teams in the mid teens age bracket. It really is life changing! After my mummy & daddy spoke to Manchester’s Willink metabolic unit (My specialists) they’ve been told other similar Gene therapies have been priced in excess of 2million pounds once approved. We haven’t got time to wait and see if the NHS (MHRA & NICE) approve this treatment in years to come because any lost skills can’t be gained back. In order to keep my quality of life I have right now, the Gene therapy is needed before any brain damage has taken place. And due to my age this puts me in a huge advantage for around 1 year. The gene therapy essentially replaces my faulty gene with a working gene through a port in the brain. I have ONE single enzyme deficiency in my body which is leading to a build up of a harmful toxic substance (Heparan Sulfate).
I really need everyone’s help & support by getting my story out there. Because if 2million people could all just donate £1, it doesn’t seem such a momentous task after all.
https://gofund.me/b857d4e43
Our £1 link:
https://www.crowdfunder.co.uk/p/qr/VlDv3RKn?utm_campaign=sharemodal&utm_medium=referral&utm_source=shortlink
