CHESTERFIELD COUNTY, Va. -- There is now new hope on the horizon for a Chesterfield girl battling a form of terminal childhood dementia.
In December, CBS 6 first introduced viewers to 9-year-old Abby Alvey, who is one of only a couple hundred children across the country with a terminal disease called Niemann-Pick Disease type C.
It’s a form of childhood dementia that currently has no known cure.
The FDA recently authorized two drugs, MIPLYFFA (arimoclomol) and AQNEURSA (levacetylleucine) that are supposed to help stop the progression of the rare disease.
The approval is giving Abby's family a whole new sense of hope.
“The sun is rising, the clouds are parting, or whatever euphemism you want to use. It’s looking really good,” said Abby's dad, Garland Alvey.
Abby and her family testified in front of the FDA to help push for the drug's approval to try to give their little girl a fighting chance at life.
Without treatment, Abby was beginning to lose her ability to walk, talk, speak, and eat.
Garland reflected on how proud he was of Abby for playing a part in helping it get approved.
“I’m not even exaggerating when I say she is saving lives,” he said.
According to Alvey, Abby has been taking one of the drugs for a month.
He said the results are already evident to her parents and her teachers. The family is receiving calls from her school, saying they have noticed a difference in her engagement.
“We also see it at home and we are trying to say maybe she is just feeling a bit better. but we are starting to see signs we can’t ignore that she is way more cognitively in the moment,” he said.
Abby’s doctor at Children’s National Hospital in Washington, D.C., applauded the work the Alvey family did in getting the medication approved.
“It’s really inspiring to work with families like hers. That advocacy does lead to change when we all work together and are on the side of these wonderful children,” said Dr. Shur.
While the potential of these drugs is giving this family a new sense of optimism, they plan to not stop working to make Abby live a lifetime in a short time.
“We know we are that much closer to a cure and now that we have drugs that stabilize and stop the progression - the next step is a cure and that was a pipe dream,” he said.
Abby’s parents created a nonprofit called Abby Strong to help raise funds and awareness for more research and support for families with kids who have rare diseases.
If you are interested in learning more about the organization, you can click here.
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