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.
A clinical trial for a potential treatment is expected to begin in the United States later this year.
Leni’s parents are urging the government to help fund the research so the trial could also include patients in the UK.
Her mother says early intervention is critical.
“Early treatment is key for these children. The damage cannot be reversed once it’s done,” she 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.
“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.”
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