Two-year-old Leni is described as a bundle of energy – a smiling toddler who loves to make people laugh., , Every new word she learns and every developmental milestone she reaches, brings her family immense joy., , But for her parents those moments are cruelly double-edged., , Because Gus and Emily Forrester know in the coming years they could watch their daughter gradually lose all her physical and cognitive abilities., , Leni has Sanfilippo disease, a rare genetic condition often described as childhood dementia., , SOURCE https://www.itv.com/news/2026-03-23/parents-in-race-against-time-to-save-two-year-old-girl-diagnosed-with-dementia Credit: ITV NewsA TWO-YEAR-OLD girl has been diagnosed with dementia which could see her lose all physical and cognitive abilities.
Her are in a race against time to get their daughter included in a clinical trial for potentially life saving .
Two-year-old Leni has Sanfilippo diseaseCredit: ITV News
Sanfilippo disease is a rare genetic condition often described as childhood dementiaCredit: ITV News
Leni, described as “a bundle of energy”, has been diagnosed with Sanfilippo disease – also known as childhood .
Her parents, Gus and Emily Forrester, say this is “every parent’s worst nightmare” as there’s no known treatment or cure.
The rare genetic condition affects around 240 children born in the UK each year.
It is caused by an enzyme deficiency that means the body is unable to break down certain molecules.
As these molecules build up, Leni’s brain cells will be irreversibly damaged.
Without treatment, it’s expected that Leni won’t live past her mid-teens as the disease progresses, making her unable to eat, drink, walk, and talk.
“Every day that passes without treatment, this toxic waste is building up in our child’s body,” Emily told ITV News .
There’s currently no treatment available in the UK.
In the , a clinical trial for experimental treatment is set to go ahead later this year, and Leni’s parents are hoping their precious daughter and other UK patients can be included.
But without funding from the UK government this isn’t possible.
They are calling for newborn screening to help detect the disease as early as possible and for the government to accelerate research for treatments with funding.
Emily said: “If she has to wait six months, that could mean she can no longer talk. If she waits 12 months, that could mean she loses the ability to walk.
There’s currently no treatment available in the UKCredit: ITV News
It affects around 240 children born in the UK each yearCredit: ITV News
“It’s extremely frustrating because the science is there. The data is there. It’s proven to be effective, and yet we cannot access it.”
Professor Brian Bigger at the University of Edinburgh has developed a gene therapy to tackle childhood dementia, which he says is an overlooked disease.
It works by delivering a missing gene into the blood stem cells – but significant funding is required to start clinical trials.
This comes as Sam Fairburn, the mother of who died in December 2024 of young onset dementia, is starting a foundation to help other families.
Young onset dementia is different to childhood dementia, but both involve progressive brain deterioration.
Young or early-onset dementia usually describes symptoms of the brain-robbing disease that strike before the age of 65.
The likelihood of developing dementia increases significantly with age, with over 900,000 Brits living with the condition.
The disease first presented when Andre turned 21. He started acting different to his usual cheeky self – repeating words and not understanding instructions.
Doctors said it was autism but the condition rapidly deteriorated as Andre started to forget words and why he’d gone to the shops.
After Sam pushed for her son to get an MRI scan, the consultant said he thought he was looking at the brain of a 70-year-old.
A cognitive test clearly showed he had dementia as he confused a crocodile for a dog and drew a squiggle instead of a triangle.
In the last few months of his life, Andre was using a wheelchair as the disease meant he was unable to walk or even stand.
On December 27, he sadly passed away in his sleep.
Now, Sam wants to start a foundation to help other families suffering with the affects of young onset dementia.
What is Sanfilippo syndrome?
SANFILIPPO syndrome – also known as Mucopolysaccharidosis type III or MPS III – is a rare neurodegenerative disease.
It causes children to lose all the skills they’ve gained (like talking and walking), suffer seizures and movement disorders, and experience pain.
Its symptoms, which worsen as kids get older, mean it is often described as causing childhood dementia or Alzheimer’s.
Sanfilippo Syndrome is estimated to affect one in every 70,000 births.
It occurs in children born with a single genetic defect that causes their body to lack a necessary enzyme.
Symptoms, which also include speech and language delays, ear infections, headaches, sleep problems and hyperactivity, usually surface between the ages of one and six.
Sadly, it has a 100 per cent mortality rate, with sufferers typically passing away in their early teens.
There is currently no cure, but clinical trials for enzyme replacement therapy and gene therapy are taking place.
Source: Cure Sanfilippo Foundation
A professor the University of Edinburgh has developed a gene therapy to tackle childhood dementiaCredit: ITV News
Eze Chidiebere Paul
I'm dedicated to creating high-quality content that informs, inspires, and delights. If you have any questions or suggestions, don't hesitate to get in touch. Happy reading!
