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Columbia Family Wants to Raise Another $2M to Cure Rare Syndrome

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2.6 from 7 votes
Monday, September 05, 2016

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CHARLESTON, United States - When Glenn O'Neill raised $2 million in online donations to cure his daughter's metabolism disorder, he wasn't satisfied.

"There are more children basically dying," said O'Neill, who lives with his family in Columbia. "These are children that we know. These children, literally if they don't get in the clinical trial, they have no chance."

An estimated one in 70,000 babies, including O'Neill's daughter Eliza, are born with Sanfilippo syndrome, an inherited disease that progressively destroys a child's ability to walk, to communicate and to eat on their own. Patients diagnosed with the syndrome rarely survive beyond their teenage years.

"You get to know your child and then all of a sudden they're taken away," O'Neill said.

Eliza O'Neill, whose story went viral a few years ago, was diagnosed when she was 3 years old. The Go Fund Me campaign set up in 2013 to raise money for Eliza so that she could participate in a clinical trial raised $2 million, setting the South Carolina record for the crowdfunding website.

Eliza, now 6 years old, was treated a few months ago with a one-time gene therapy injection at Nationwide Children's Hospital in Columbus, Ohio. The procedure only lasted 30 minutes, but it required the O'Neill family to remain quarantined for about 2 years to ensure that Eliza didn't contract a specific virus. Otherwise, she would have been disqualified as a viable candidate for treatment.

"We did not come in contact with other people," Glenn O'Neill said. "We knew that this trial would be her only chance at life."

In May, Eliza became the first child diagnosed with Sanfilippo syndrome who received the gene therapy injection. It isn't covered by health insurance.

"If it works like it did in the mouse model, it's basically a cure," O'Neill said.

Already, he has noticed a difference in his daughter. She's starting to use words again, little by little.

"She's been able to stop and engage," he said. "There's just a new presence about her that's hard to explain."

That's good news for the O'Neill family, but Glenn isn't stopping there. He wants to raise another $2 million through the family's Cure Sanfilippo Foundation so that other children may access treatment during this experimental stage.

"We've got to save these kids," he said.

For more information about Sanfilippo syndrome or to donate money, visit the Cure Sanfilippo Foundation's website, curesff.org.

Author: Lauren Sausser
Source: The Post and Courier
2.57142857143
2.6 from 7 votes
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