Just4Children are fundraising to help Tate access specialist medical treatment for Sanfilippo Syndrome Type A, including gene therapy where clinically appropriate, together with associated medical expenses and support for his ongoing needs.
My son, Tate, has Sanfilippo Syndrome Type A (MPS IIIA), a rare, life-limiting neurodegenerative disease, often described as childhood dementia. This devastating condition progressively takes away children’s ability to communicate, learn, walk and eat independently, and significantly reduces their life expectancy.
But right now, Tate is an active, happy little man. He runs, climbs, eats by mouth and enjoys life. His mobility is great, and he currently needs no pain medication or medical intervention to support his feeding or mobility. Looking at him, you would not see the devastating disease threatening his future.
That is why I am fighting so hard to get him treatment now, while he still has so much to protect.
On 17 September 2026, Fayuvi (formerly known as UX111) gene therapy received approval from the US Food and Drug Administration (FDA) for the treatment of certain children with Sanfilippo Syndrome Type A. This is an enormous milestone, bringing renewed hope for Tate’s future.
The next challenge is accessing and funding treatment. The estimated treatment cost is £2.95 million, with an additional £100,000 potentially required for associated medical expenses, assessments, travel, accommodation and follow-up care. This brings our fundraising target to £3,050,000.
These costs are estimates and will need to be confirmed by the relevant medical providers.
This campaign aims to give Tate the opportunity to access Fayuvi, subject to medical assessment, eligibility and availability, or another appropriately regulated and clinically recommended disease-modifying treatment that may help slow the progression of his condition.
The primary purpose of this campaign is to fund treatment and reasonable costs directly associated with accessing it, where clinically appropriate and approved by Just4Children. These may include:
Time matters. Tate is doing amazingly now, but Sanfilippo is progressive, and unfortunately, a great deal can change within a year. My goal is to raise this money in time for him to receive treatment while he still retains so many of his abilities.
If sufficient funds cannot be raised in time, or Tate’s medical circumstances change so that disease-modifying treatment is no longer available or clinically appropriate, we would wish to use the funds raised to support his ongoing medical, therapeutic and specialist care needs, subject to Just4Children’s charitable objectives, fundraising terms and trustee approval.
In those circumstances, support could include clinically recommended physiotherapy, hydrotherapy, occupational therapy, speech and language therapy, specialist equipment, communication aids, mobility equipment, respiratory support and other appropriate interventions intended to maintain Tate’s comfort, safety, independence and quality of life.
Any proposed expenditure, including treatment and alternative care arrangements, would be subject to appropriate supporting medical evidence, the availability of sufficient funds and approval by Just4Children in accordance with its charitable purposes and funding policies.
As medical research continues to develop, treatment options and pathways may change. Our priority remains giving Tate the opportunity to access appropriate medical treatment and the specialist support he needs.
I’m not a medical professional or expert. I’m just Tate’s mum, a solo parent doing everything I possibly can to give my little man a chance at a future that this disease is trying to take away from him!
There is still a very long way to go, but every donation, every fundraiser and every share helps me get closer.
From the bottom of my heart, thank you for standing beside me and giving Tate hope.
Tate & Tammy
