Fighting Rare Disease: One Family’s Inspirational Journey
Prevention & Treatment The Gray Family stands up to Batten disease, modeling bravery for the many Americans fighting rare disease.
Five-year-old Charlotte Gray loves Mickey Mouse and the movie “Frozen,” but she and her family are facing a difficult diagnosis. Charlotte and her little sister, two-and-a-half-year-old Gwenyth both have Late Infantile Batten disease, a rare genetically inherited disorder that results in progressive neurological impairment including seizures, blindness and dementia.
The girls’ parents, Kristen and Gordon, a Hollywood movie producer known for movies like “The Rookie” and “Invincible,” remember getting the grim news from the doctor. “It was rare, fatal and there was no cure,” Gordon says. “That’s basically all the information we got, aside from a pamphlet with information on the disease.”
Raising money and awareness
Life expectancy for both girls ranges from age 6 to 12, and Charlotte is losing some of her speech and motor skills. Gene therapy is still in development, and is expensive.
“The diseases are so rare and by the time you get around to raising the money to find a cure to stop the progression of the disease, it’s too late.”
“We’ve had to generate a lot of attention so we could raise the money and, really from scratch, fund the treatment,” says Gordon. “We were starting from zero.”
The Grays started the Charlotte and Gwenyth Gray Foundation to raise the $10 million needed to pay for urgent medical research to treat Batten disease. They’ve raised $3 million in six months through social media and four awareness events. Stars like Rihanna, Jennifer Garner and Channing Tatum have supported the effort.
Funding the research
“The rare disease community is definitely coming together because there isn’t enough attention on these diseases,” says Kristen, who hopes the government will fund rare disease research. Treatment is tough in no small part because there are fewer than 400 FDA-approved treatments.
“The diseases are so rare,” Gordon explains, “By the time you get around to raising the money to find a cure to stop the progression of the disease, it’s too late. The Grays are grateful for the awareness and financial support they’ve received.
Adds Kristen: "We believe we’re going to find a cure to stop this horrible disease and save our children, and hopefully pave the path for other families and children after us.”