A 4-year-old girl’s family is desperately trying to raise $2.5 million to produce the treatment for the rare disease that will shortly start eating away at their daughter’s body and mind.
In July, Eliza O’Neill was diagnosed with Sanfilippo Syndrome, an extremely rare genetic disorder that is caused by an inability to break down certain sugars. Her family is now racing against time to cure the disease that will soon begin showing its debilitating effects.
Only an estimated 2,000 children are born with Sanfilippo Syndrome each year worldwide. The children who are diagnosed with it typically develop deficiencies in muscle growth, speech and brain functions by age five; most are not expected to live into their early 20s.
Many of the victims are confined to wheelchairs as the disease begins to spread.
“She does stuff like any active child,” said Eliza’s father Glenn, who has said that his daughter is not aware of what will soon start happening to her. “It’s hard to believe this will come as quickly as it does, but other parents have said yes, it will.”
Six months ago, the family began raising money through fundraisers and 5k events in their Columbia, S.C. community. They have currently raised about $250,000.
This, however, is a mere fraction of the total cost of producing the treatment and running the clinical trial, which is estimated to be about $2.5 million.
“We realized we would fall short,” Eliza’s father, Glenn, said. “This means everything to us.”
The family turned their attention to a wider audience: they decided to make a viral video about their cause. A Google search on “how to make a viral video” led the family to filmmakers DL Cade and Benjamin Von Wong, who produced the film.
“Be part of the miracle: Saving Eliza” went online Wednesday morning. In the video, Eliza is seen with 11-year-old friend Izzy Jurado, who also has the disease. Izzy has trouble moving around and controlling her body.
The video ends with a plea: “Please share. Please share.”
The trial will be conducted at Nationwide Children’s Hospitals in Columbus, Ohio. The hospital is working to get FDA approval to start treating the disorder. Researchers have said that they can begin ordering doses of the specific gene that the patients are missing as soon as $1 million is raised. The treatment will cover 15 children, who will be injected with the gene and monitored for the next 15 years.
“We have the cure. It’s going to happen at some point. But it won’t happen without money and if it gets delayed, Eliza won’t get it and a lot of other kids won’t get it,” Glenn continued.
The family is collecting contributions at GoFundMe.Com. All contributions will go directly to the funding the trial.