WHAT IS batten disease?
Batten Disease is a rare neurodegenerative disease causing seizures, vision loss, cognitive impairment and early death. There is no cure... yet. Parents are forced to make excruciatingly painful decisions as the disease takes its course. Eventually, these families are left to face life without their child.
How common is batten diseasE?
Every year, hundreds of children are born with some variant of Batten Disease, all of which are fatal and have no cure. Worldwide, roughly 14,000 children are afflicted with this disease. Siblings also have a 25% chance of developing the disease, which makes the diagnosis all the more devastating for families as they wonder the fate of their other children.
the science behind batten
In Batten Disease, a simple gene mutation prevents cells from breaking down fats and proteins, leading to a build-up in the cells' trashcans, or lysosomes. This results in cell death and the body’s inability to function normally. The condition is autosomal recessive, meaning both parents must carry the same abnormal gene in order for their children to develop the disease. There are fourteen known variants of Batten - CLN1, CLN2, and so on - and each is unique enough to require its own research and trials. We are focused on Mila’s variant, CLN7.
As a result of the work we initiated toward a gene therapy, there is now a CLN7 Natural History Study underway. The idea is to collect data from children to better understand the disease and help with treatments. If you are interested in enrolling your child, please email me at firstname.lastname@example.org and I will connect you with the study coordinator.
IS THERE A TREATMENT?
There is no cure for Batten Disease... yet. However, we are working hard to start and fund new treatments that could help children with Batten and other devastating neurological disorders. Our work is for Mila and for the millions of other children like her that deserve a chance.
Learn more about Our Work.
We need your help now
Mila has lost her vision and much of her ability to walk and talk. But she is a fighter. We need your help now to give children like Mila a second chance at life.
Mila's Miracle Foundation, Inc. is a 501(c)(3) nonprofit organization. All donations are tax-deductible.