A two-year-old girl faces the prospect of losing her physical and cognitive abilities following a devastating diagnosis of a rare genetic disorder.
Gus and Emily Forrester spoke to ITV News on Monday about their daughter Leni's condition, Sanfilippo Syndrome Type B, describing it as "every parent's worst nightmare".
The illness, formally known as Mucopolysaccharidosis type III, is a terminal neurodegenerative disease commonly referred to as childhood dementia.
According to the Cure Sanfilippo Foundation, affected children typically begin displaying developmental delays between the ages of one and six, and no approved treatment or cure currently exists for the condition.
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Gus and Emily described Leni's condition as every parent's worst nightmare
|GOFUNDME
"All your dreams for your child's future are taken away," Emily told ITV News. "To be told that she has this condition, and there is no treatment, no cure and no support — It's completely earth-shattering."
Emily explained time is of critical importance for children with this condition, as without intervention, she explained, her daughter's body will become overwhelmed with toxic substances, causing irreversible damage.
"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," Emily said, expressing profound frustration at the barriers preventing access to promising treatments.
"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," she added.
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The Forresters are now urging authorities to permit British patients to participate in a clinical trial for a potential treatment anticipated to commence in America later this year.
The family has established a fundraising campaign to support potential treatments for Leni and cover medical expenses that could enhance her quality of life.
Their GoFundMe appeal has garnered remarkable support, with donations surpassing $250,000 (£186,000).
The Forresters have pledged to share the proceeds with institutions providing vital assistance.
"We will also donate funds to Great Ormond Street Hospital, who are providing Leni with truly world-class care, and Cure Sanfilippo Foundation, which is the only charity dedicated to Sanfilippo Syndrome and has been a huge support for us and many other children and families," they wrote on their fundraising page.
"If we can't get treatment, she will suffer the most awful physical and mental decline you can imagine and then die in her early to mid-teens," Emily said.
Gus added his own stark assessment of their daughter's prospects.
"As parents, your role is to protect your children and provide every opportunity you can," he said. "Without any treatment, her future and her reality is very, very dark."
Gus and Emily want the government to permit British patients to partake in a clinical trial for a potential treatment
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Beyond their personal battle, the couple have launched an Instagram account to raise awareness of childhood dementia and connect with families facing similar circumstances.
They are also advocating for newborn screening programmes to identify rare genetic conditions at the earliest possible stage.
Professor Brian Bigger from the University of Edinburgh, who has pioneered a gene therapy approach to the disease, stressed the necessity of state funding.
"Charities typically can't fund this kind of thing," he saud. "There are hundreds of kids like Leni out there who don't have any therapy, and these treatments have the potential to be transformative."
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