Twelve months ago this family had their life turned upside down.
Pam and Chris Andrews’ were told the news both of their daughters have a rare, fatal genetic disorder referred to as “childhood Alzheimer’s.” ( Niemann-Pick disease type C1)
Most children diagnosed with the disease are dead within 10 years of diagnosis, Dr. James Gibson, a geneticist at Ascension’s Dell Children’s Medical Center in Austin, Texas, told them, shares PEOPLE.
Six-year-old Belle was diagnosed on March 10, 2016. Three weeks later, Abby, now 2, was diagnosed. Both Chris and Pam, who met on Match.com in October 2006, inherited an autosomal recessive gene and both girls inherited the genes from them. The girls had a 25 percent chance of inheriting both genes from their parents.
“We were facing losing our entire family,” says Pam, 44. “There was no more normal,” says Chris, 45. “Nothing was ever going to be normal again…We were crushed.”
Though devastated, the Andrews quickly rallied and found a promising clinical trial for the disease. The drug VTS-270, made by Vtesse, Inc., is not a cure but treats the symptoms of the disease, which include gradually losing the ability to think, hear, walk, talk and eat on their own.
They also decided to start the Firefly Fund, a foundation to find a cure for this and other rare, neurodegenerative diseases that affect children.
NPC1 is a progressive, neurodegenerative disease where the lysosomes of all cells of the body accumulate toxic amounts of cholesterol, causing progressive damage to the brain, spleen, liver, and other tissues.
If left untreated, Belle and Abby would likely eventually suffer from seizures, dementia, ataxia, and cataplexy. As the disease progresses, children diagnosed with NPC1 require feeding tubes because of difficulty swallowing, are confined to wheelchairs, are unable to speak or recognize loved ones, and die in adolescence.
“We will not let them die,” vows Pam.
Adds Chris: “No matter what we have to do, no matter who we have to call, no matter how much money we have to raise — whatever it takes — nobody’s going to take my kids.”
This is so heartbreaking. My own mother had early onset dementia. She was finally diagnosed in her 50s. That was hard enough. I can not fathom a young child going through the same.
We wish them all the best and hope that new research finds a cure for these young girls before it’s too late.
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