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Drugmakers Are Focusing On Rare Disease Treatments To Help Patients Like This Brave Little Girl

Mar 27, 2015
When it comes to saving your child's life, there's no obstacle you wouldn't overcome. That was the O'Neill family's response when they learned their daughter Eliza was born with a rare and fatal genetic disorder called Sanfilippo syndrome.

Sanfilippo presents itself in young children who appear healthy at first but then start regressing intellectually. There is no cure, and most patients don't make it past their teens. Clinical research trials are the last hope for many families, but having the funding necessary to start trials is a challenge.

Through sheer determination, the O'Neills collected $2.2 million in donations for research of Sanfilippo syndrome. Here's hoping it works out for them and millions of others.
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