GoFundMe record broken as strangers raise money for 4-year-old girl's rare disease

GoFundMe record broken as strangers raise money for 4-year-old girl's rare disease

Eliza suffers from the more serious type of Sanfilippo Syndrome.

Eliza O’Neill was a normal 4-year-old girl, until she got a shattering diagnosis. Eliza’s parents were told she has a fatal disease known as Sanfilippo Syndrome. With no cure, her parents have started an online fundraiser to raise $1 million for a gene therapy trial. Scheduled for late 2014, this gene therapy trial offers hope for Eliza and other children like her.

According to the fundraising site, Sanfilippo affects boys and girls of all races, and is found all over the world. This rare disease generally kills the patient by their teens, sometimes sooner if the case is more severe. Eliza, unfortunately, suffers from the more serious type of the disease. By age 5, she will lose the ability to speak and, by 6, she will likely have irreversible brain damage.

WACH Fox reports that the parents have raised more than $814,000 via GoFundMe, setting a new fundraising record. The previous GoFundMe record was held by Boston Marathon bombing victim Jeff Bauman. They will need to raise another million prior to the start of the trials.

The disease is already attacking Eliza’s metabolism and preventing her body from breaking down sugar molecules. Nationwide Children’s Hospital in Columbus, Ohio, will conduct the gene therapy trial.

Sanfilippo Syndrome is described by Medline Plus as a metabolism disorder passed down through families. It makes the body unable to properly break down long chains of sugar molecules. Sanfilippo type A is the most severe form. Persons with this type are missing or have an altered form of a critical enzyme. Sanfilippo type A generally has the worst prognosis.

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