By A. Pawlowski
Eliza O'Neill's parents call it their miracle story. After years of
fundraising, 726 days of self-imposed isolation and non-stop
determination to save their 6-year-old daughter's life, she has finally
become the first patient in the world to receive an experimental gene
therapy for her deadly disease.
"We have tons of hope for Eliza now. So much more hope," Glenn
O'Neill, her dad, told TODAY. "I never used to think about the future,
because I didn't want to. But now, I do think about it."
Eliza has Sanfilippo syndrome,
a rare disorder that affects the brain and progressively robs children
of language and understanding. Some call it "childhood Alzheimer's."
Most victims eventually become immobile and don't live beyond their
teens. Eliza has type A, the most severe form of the disease.
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Source: NBC News/TODAY (Health and Wellness)
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