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Parents of Child Suffering From Sanfilippo Syndrome Raise Over $1 Million

First Posted: Jun 04, 2014 06:48 AM EDT

Eliza O'Neill is a four-year-old from South Carolina who suffers from Sanfilippo Syndrome-Type A, which is a rare degenerative disease that eventually disables someone's ability to speak or walk as it progresses.

Sanfilippo Syndrome has no cure and type-A is the most severe form of the disease. Most of those diagnosed don't live far into their teens. Months ago, Eliza's parents, Glenn and Cara, decided to make a video about Eliza in hopes of raising money to research the rare untreatable and incurable disease. The video eventually went viral and they raised over $750,000.

But in order for the fundraising to be worthwhile, the video needed to raise $2.5 million to help fund two researchers at the Nationwide Children's Hospital in Columbus, Ohio. Doug McCarty and Haiyan Fu have been working on a cure for Sanfilippo syndrome for over 16 years and the next step in their studies is setting up a clinical trial for their gene therapy treatment (which they found works in mice.)

"We need to fund this clinical trial portion. Thanks to donations from around the world, we are funding the medicine production," said Glenn O'Neill in this CBS Local article. "The final step is $1 million more to fund the clinical trial and we are looking to raise that before October. We can't have money be a limiting factor in not keeping this clinical trial on schedule."

Prior to April, the video generated over $750,000, and since then it added an additional $777,000 for the cause. Funding for the disease is not at all plentiful since all forms of Sanfilippo Syndrome affect 1 in every 70,000 births, which equals about 2,000 children worldwide. Diseases this infrequent typically don't get good funding because there aren't well-known organizations that are seeking donors for research purposes. And most investors/donors aren't willing to sink millions of dollars into a disease for which so little is known.

But the O'Neill's are on their way, and $1 million short of their goal thanks to GoFundMe.com where donations are accepted. In fact, they're only $31,000 away from breaking the GoFundMe record (they've raised $777,000 since registering on the website). If the donations continue at the rate they've been, Eliza will hopefully be treated before it's too late.

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