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 affected by neurodegenerative diseases access to specialized multidisciplinary care, all in one place, during a single visit to our clinic. At the same time, our expert team of clinicians are simultaneously collecting critical research data to help better understand these diseases and eventually inform future treatment paths.

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, collaborative programming initiatives, and grant funding to labs working on biomarker discovery, data collection and published findings. Our hope is that the more we learn about this devastating childhood condition, the more effective treatments will be in the future.