A girl with a rare genetic disorder celebrated her eighth birthday this week – and with a life expectancy in the early teens, every year counts.
Eliza O’Neill suffers from Sanfilippo syndrome, a condition known as childhood Alzheimer’s where toxic cells affect the brain resulting in dementia, loss of speech, blindness and eventual death.
She was the first patient in a new gene therapy clinical trial last year that her parents considered her only hope at maintaining motor functions and speech, as there is no cure for the condition.
Though she has lost the ability to speak, Eliza is still learning new skills and her birthday has sparked a campaign to raise $100,000 in 10 days to fund further research.
