(NEW YORK) -- The O’Neill family has been racing the clock to fund a cure for their 4-year-old daughter’s rare, terminal disorder, and in the process, they’re on track to break a fundraising record on GoFundMe.com. Since early April, the O’Neills have raised more than $777,000 toward a cure for Sanfilippo syndrome, the deadly genetic disorder that’s now barely detectable in their daughter Eliza.
It means she lacks an enzyme to break down heparin sulfate, which naturally occurs in cells, causing it to build up over time, causing a variety of medical problems. The result is that Eliza will lose the ability to speak by her 5th birthday, the O’Neills say, and she’ll die before she reaches her 20s. “The O'Neill family's efforts are a true testament of two parents' unwavering love for their daughter, and the willingness to do whatever it takes to reach their goal,” GoFundMe CEO Brad Damphousse told ABC News.
All forms of Sanfilippo affect one in 70,000 births, according to the National Institutes of Health. Because funding for rare diseases is hard to come by, parents often wind up spearheading fundraising efforts.
Researchers Doug McCarty and Haiyan Fu of Nationwide Children’s Hospital in Columbus, Ohio, have been working on a cure for about 16 years. They’ve found a gene therapy treatment that works in mice, and they hope to try it in humans. But setting up a clinical trial takes money –- about $2 million that they don’t have.
Desperate to help his daughter, Eliza’s father, Glenn O’Neill, said he Googled “how to make a viral video” and found filmmaker Benjamin Von Wong. Von Wong agreed to shoot and launch a video about Eliza to promote a GoFundMe fundraiser toward Sanfilippo research.
Now, they are about $31,000 away from breaking the GoFundMe record, which stands at $808,845 for Boston Marathon bombing victim Jeff Bauman. Celeste and Sydney Corcoran, two other Boston Marathon bombing victims, are in second place with $794,335.
The O’Neills announced Tuesday that the money raised so far is going toward making the drugs that will be used in the clinical trial, but they will still need to raise another $1 million this summer and fall to make it a reality by the end of the year.
“The first major step towards saving our daughter is complete,” the O’Neills said in a statement. “The medicine will be ready in December and Eliza and others like her will have the chance to get the treatment they so desperately need.”
Damphousse said GoFundMe’s medical category is its most popular.
“The most successful fundraising campaigns are those that evoke strong emotions,” Damphousse said. “People are far more likely to support someone they know -- especially when an individual's well-being is on the line. Accordingly, GoFundMe campaigns related to medical emergencies, illnesses or accidents have always raised more money in less time than other, less urgent causes.”
Glenn O’Neill told ABC News that Eliza sometimes gets extra attention because of the campaign, but she doesn’t understand why. Since she’s shy, she covers her face when people recognize her from the viral videos. Overall, she's doing well, he said.
“We do notice things related to the syndrome in the way that she processes information,” O’Neill told ABC News. “She is not regressing on anything though and still learning -- just at a slow pace and behind her peers.”
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