Their four-year-old daughter has a genetic disorder known as Sanfilippo disease and needs money to help fund clinical trials for experimental treatment that could save lives.
Time is of the essence for the O’Niell family, who are desperately trying to raise funds that could help save the life of their daughter. Four-year-old Eliza suffers from a rare genetic disorder that will claim her life if a cure is not found.
The disorder is known as Sanfilippo syndrome. It is a metabolic disorder that occurs when a person’s body lacks the enzyme needed to break down, heparane sulfate, a lengthy chain of glucose molecules found in cells. Complications from Sanfilippo syndrome include delayed development and learning that eventually slows and begins to deteriorate. According to her family, Eliza will no longer be able to talk within the next year and she could die as early as her teenage years.
Research on this rare disorder has been underway for nearly the last two decades. To date, no specific treatment is listed for Sanfilippo syndrome. Hope is available to the O’Niell’s and similar families in the form of two scientists, Doug McCarty and Haiyan Fu, who have been working out of the Nationwide Children’s Hospital in Columbus, Ohio. They have developed a treatment that has proven successful on lab mice. It is a form of gene therapy that they wish to move into clinical trials in order to test on humans.
However, funding is not readily available, especially considering that Sanfilippo syndrome, in all its forms, only affects about one out of 70,000 children born. It was because of the rarity of the disease, and their love for Eliza, that the O’Neills decided to take on the burden of fundraising themselves.
Using online searches, Glenn O’Neill, Eliza’s father, found a filmmaker who would help them produce a viral video meant to raise funds through GoFundMe, an online crowdsourcing site. The filmmaker, Benjamin Von Wong, stayed with the family and made a video that did indeed go viral.
So far it has helped raise over $770,000.
The collective efforts have raised so much that they are close to breaking the GoFundMe site’s record for funds raised. The current top two spots are held by three victims of the Boston Marathon bombing.
But the family is far from their own goals. The money they have raised thus far is being used to produce the medication needed for the clinical trials. The O’Neills state that they will need to garner another $1 million in the next few months to see the trials come to fruition.
Leave a Reply