Eliza O’Neill is racing against the clock. The 3 year old was diagnosed with Sanfilippo syndrome – the most severe form of the genetic debilitating disorder. The clinical-trial costs are an overwhelming $2.5 million. And her parents, undeterred by the challenge, have rallied internet supporters and already broken online fundraising records. Read on to see how the campaign came together and hear from Eliza’s little brother.
Her mother, Cara, 39 said:
After we were told what was wrong with her, we held each other and cried in the parking lot. But we knew we could not let our daughter die.
Cara and her husband, Glenn, found out that researchers at the National Children’s Hospital developed a form of gene therapy that they believe could be a cure for the syndrome. The researchers had success testing a cure in mice, but lacked funding for clinical trials on children. The O’Neill family hopes to raise the $2.5 million to fund a trial in which Eliza, as well as several other children, will be able to participate.
About 1 in 70,000 children is born with Sanfilippoo syndrome – also known as MPS III. According to the National MPS Society, children with Sanfilippo syndrome are missing an essential metabolic enzyme that prevents the body from properly breaking down sugar and causes nerve damage, brain damage, blindness and eventually death.
As the video above notes, Eliza will likely stop walking in the next 2 years, and lose the ability to speak by 5 years old. She will also develop seizures as the disease progresses untreated.
But Eliza’s outcome could be very different thanks to the record-breaking support of strangers around the world.
Here is a clip showing how the #SaveEliza campaign came together.
Dad, Glen, 41 said:
It’s all from extraordinary people donating whatever they can afford. People have reached out saying that they’re out of a job but they want to donate $10.
The O’Neill’s have held numerous events to fun raise including dance-a-thons and golf tournaments. But the vast majority of financial support has come in from online support due to two videos – the one above and the one seen below featuring Eliza’s 7 year old brother.
Eliza’s older brother is helping care for his sister too. Glenn explains:
She only lets her 7-year-old brother Beckham put her to sleep at night. We peek in and see that he gets into bed with her, holds her hand and talks to her until she falls asleep.