Dec. 8—Most people haven't heard of the CLN2 Batten Disease, an illness that ravages the motor skills, speech and vision of its host. Those who have the illness — mostly children — have a greatly shortened life expectancy.
Hopefully, this rare disease is getting more publicity of late after a report over the weekend by the Herald's Pamela Knudson, who reported on the sad story of young Grand Forks resident Daphne Enger.
Enger, just 6 years old and diagnosed at age 3, is fighting Batten Disease, an illness that has no cure but one that can be treated to slow the frightening effects.
In the Herald report, Daphne's parents, Brice and Lindsey, ask readers to sign a petition that urges the Food and Drug Administration to approve gene therapies for children with the disease. The idea, Lindsey Enger said, is to "get the attention of the FDA" to "save our children."
The Engers stress that signees do not have to donate, but can simply add their name to a growing list of people concerned about the fight that little Daphne and others like her are enduring.
Those interested in signing the petition can find it online at https://chng.it/wRZjqXZk9G. It only takes a moment to do it.
As reported by Knudson, parents of children with Batten Disease want to fast-track potential therapies that can be used to treat it. And, if enough people sign the petition, the FDA might be persuaded to move the therapy forward.
"We need the FDA to see that these children's lives have potential, they have meaning," Lindsey Enger said in the Herald report. "(These children) have the right to live the same full and long life everyone else is promised. These children, including Daphne, deserve to live. ... We need to give our children every possible chance to save their lives."
Those who do choose to sign the petition can leave comments. They'll also see that while Batten Disease is rare, the Engers aren't alone.
Wrote one woman: "My son Daniel, who had CLN2, didn't get the chance to participate in compassionate use of the enzyme replacement clinical trial. My friends' children deserve the chance to participate in a gene therapy trial if there is one available. CLN2 doesn't give the luxury of time to wait."
As of Monday morning, 9,298 people had signed the petition, with a goal of reaching 10,000. Perhaps Grand Forks-area residents who read of the Engers' plight will consider adding their name to the growing list.
"This is not the life that our children deserve to live. There's better treatment for our children, and that's gene therapy. We have rights as parents and our voices need to be heard," Lindsey Enger wrote on the petition website. "To be able to have gene therapy offered to our children, would allow many children to live a normal life."