Family faces uncertain future as shutdown stops life-saving research for rare disorder

(NBC News) - Three-year-old Eliza O'Neill, of Columbia, South Carolina is facing an uncertain future as a federal agency helping to find a potentially life-saving cure for the rare disorder closed due to the government shutdown.

Eliza was diagnosed with Sanfilippo Syndrome Type A, a rare genetic disorder which doesn't allow the body to break down certain enzymes. There is no cure for Sanfilippo Syndrome Type A which has a life expectancy in the teens.

Gene therapy research at a children's hospital in Ohio has cured the disease in mice, and they soon hope to study the cure in humans. However, due to the government shutdown the research has stopped.

Since Wednesday night's report more than $5,600 in donations have rolled in to help fund the research.

"These kids by age six are in very bad shape," said Glenn O' Neill, Eliza's dad.

With little time to spare, the O'Neills joined other families and got involved raising money to fund the final, critical phase of the clinical trial for San Fillippo Syndrome Type A. The group hopes to raise $2.5 million over the next nine months to fund the final phase of the research in order to take it to human testing.

"They think in terms of years," said Eliza's mother Cara O'Neill. "We don't have years. We think in terms of months and weeks."

Glenn said they don't know how long they will have with Eliza but things can happen quickly.

"I kiss her hand, with my lips to the back of her hand, every night and just tell her we love you forever because someday she may not be able to understand what I'm saying... without any treatment," Glenn said.