GASTON COUNTY, NC (WBTV) - Many of you know Abby Ferguson. For those who don't, here's a quick summary:
- 11-year-old in Dallas, North Carolina.
- Infectious smile.
- Beautiful spirit.
- Lives with the rare and tragic Sanfilippo Syndrome.
- A genetic, deadly, progressive disorder that affects the brain.
- Though Abby is 11, she has the mind of a 5-6 year old.
- She was diagnosed three years ago.
- Her health and disease have remained stable since then.
- Long-time #MollysKids.
Her family has been fighting and fighting and fighting to get a clinical trial going for kids with Sanfilippo.
So this is huge news from Abby's mom Wendy:
"Thanks to so many selfless, kind-hearted supporters we, along with many other affected families, raised enough funds to get the trial started at Nationwide Children's Hospital in Ohio," she said.
The Gene Therapy clinical trial already started. The easiest way to describe Gene Therapy is a one-time injection of normal genes to replace the defective ones. It was most recently used in a clinical trial for Batten Disease.
Wendy calls it "true miracle treatment".
"This Sanfilippo Syndrome clinical trial will start with nine children," Wendy said. "We do not know if Abby will be included but we are hopeful this will be the answer to ours and so many other parents' prayers."
Meantime, Abby is doing really well. She maintains her skills. She's finishing up 5th grade at Costner Elementary School and will be at William C Friday Middle School next year.
"We owe so much to so many," Wendy said. "Friends and family have contributed to funding this research, as have strangers. We can't thank everyone enough for their support."
Clinical trials are tough because you never know how they'll work.
A lot of hope gets wrapped up.
But it's a chance. A shot. And if your child lived with a disease that gave them a life expectancy of 10-20 years -- like what Abby has been told -- I can bet you'd be as thrilled as the Fergusons with any chance at all.
I love this picture of her. Feel free to come back and look at it a few times today, just as perspective on what really matters.