Our work
Our mission is to find and fund paths to a cure for devastating neurological conditions like Batten disease. We are focused on helping to grow the new field of individualized medicines which Mila’s story has opened up, and making it accessible to many more across all rare diseases. Our hope is that our work will give other children like Mila a second chance at life.
Here’s how we are accomplishing our mission…
Mila to Millions
We have the science and technology to make more treatments like Mila's possible. We have millions of children who could benefit from them. But we don’t have the framework in place to scale these treatments in time for those who need them most. We're working with academics, clinicians, regulators and industry leaders to solve this problem so we can make individualized medicines routine and accessible for many more children before it’s too late.
individualized medicines
Our foundation partnered with Boston Children’s Hospital to start the first-ever individualized medicine, called Milasen. Led by the pioneering scientist/neurologist, Dr. Timothy Yu, this Antisense Oligonucleotide (ASO) treatment which Mila received for three years targeted her individual disease-causing mutation. We are now working to turn Mila's story into an impactful treatment platform applicable across rare diseases as a whole.
New England Journal of Medicine
New York Times
GENE replacement THERAPY
Together with renowned gene therapist Dr. Steven Gray, our foundation began the work on a novel gene therapy direct to the brain for children with Mila’s type of Batten Disease, CLN7. This promising approach replaces the broken gene with the correct one in a one-time shot into the cerebrospinal fluid. The trial was developed in partnership with UT Southwestern, Aashi’s Hope and Batten Hope and launched in Spring 2021.
New York Post
Today Show
Neurodegenerative Disease Clinic
Our foundation launched the Batten and Neurodegenerative Disease Clinic at Children's Hospital Colorado with the purpose of providing families with children with neurodegenerative diseases specialized multidisciplinary care all in one place on one day. Simultaneously, research data is collected to help better understand these diseases and eventually inform future treatment paths.
About the Program
BASIC SCIENCE
In order to improve treatments for Batten disease, our foundation is dedicated to better understanding the basic science behind this condition. We support scientists working to crack the CLN7 gene through meetings with researchers and physicians, and grant funding to labs working on biomarker discovery and data collection. Our hope is that the more we learn about this devastating childhood condition, the more effective treatments will be in the future.
The Journal of Clinical Investigation
WHAT IS BATTEN DISEASE?
Batten Disease is a rare neurodegenerative disease which causes seizures, and robs seemingly healthy children of their vision, ability to talk, walk and eat, of their cognition and eventually their lives. 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. Families in this community are starting and funding new genetic treatments which offer great promise that one day soon we will be able to stop this devastating disease.
mila’s miracle foundation
Mila's Miracle Foundation to Stop Batten was formed in December 2016 upon learning our daughter Mila had Batten Disease: a fatal, devastating genetic disease. We now realize that Mila’s life has a greater purpose than we had ever imagined when she was born. She isn’t just our sunshine. She is a little ray of light, a little spark, for everyone. Our hope is that her spark will ignite others, and one day soon there will be a bright light where there was once just darkness.
WE NEED YOUR HELP NOW
Mila was given an unprecedented second chance at life through the first-ever truly personal medicine, Milasen. Now we need your help to pave this path for many more children like Mila across hundreds of rare diseases.
Mila's Miracle Foundation, Inc. is a 501(c)(3) nonprofit organization. All donations are tax-deductible.