Mila's Miracle Foundation

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. INDIVIDUALIZED MEDICINES We 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. GENE REPLACEMENT THERAPY Together with renowned gene therapist Dr. Steven Gray, we 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. NEURODEGENERATIVE DISEASE CLINIC We 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. BASIC SCIENCE In order to improve treatments for Batten disease, we are 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.