Most people have not heard of CLN2 Batten disease, a disease that ravages the motor skills, speech and vision of its host. Those with the disease – mostly children – have a dramatically shortened life expectancy.
Hopefully this rare disease is getting more publicity lately after a report this weekend by Pamela Knudson of the Herald, who reported the sad story of young Grand Forks resident Daphne Enger.
Enger, just 6 years old and diagnosed at age 3, battles Batten’s disease, an incurable but treatable condition to slow its 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 point out that signers are not required to donate, but can simply add their names to a growing list of people concerned about the fight 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 so.
As Knudson reported, parents of children with Batten disease want to accelerate 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, that they have meaning,” Lindsey Enger said in the Herald report. “(These children) have the right to live the same full and long life that everyone is promised. These children, including Daphne, deserve to live. … We must give our children every possible chance to save their lives.
Those who choose to sign the petition can leave comments. They will also see that although Batten disease is rare, Engers are not alone.
One woman wrote: “My son Daniel, who had CLN2, did not have the chance to participate in the compassionate use of enzyme replacement clinical trial. My friends’ children deserve the chance to participate in a gene therapy trial if one is available. CLN2 does not give the luxury of waiting.
As of Monday morning, 9,298 people had signed the petition, with a goal of reaching 10,000. Perhaps Grand Forks-area residents who have read about the Engers’ plight will consider adding their names to the growing list.
“This is not the life our children deserve to live. There is a better treatment for our children, and that is gene therapy. We have rights as parents and our voices need to be heard,” wrote Lindsey Enger on the petition website “Being able to provide gene therapy to our children would allow many children to lead normal lives.”