Our work

Our mission is to find and fund paths to a cure for Batten Disease and other devastating neurological conditions. Families in this community have been pivotal in raising the necessary funds and awareness. Our hope is that together with our incredible scientists and partner organizations, our work will give Mila and many others like her a second chance at life.

Here’s how we plan to do it…


ASO TRIAL

Our foundation started an exciting new Antisense Oligonucleotide (ASO) treatment which Mila is currently undergoing. Pioneered by the incredible scientist and neurologist at Boston Children’s Hospital, Dr. Timothy Yu, this first-ever fully customized treatment targets Mila’s disease-causing mutation. See the animation below to learn more on the drug, affectionately named Milasen. We are now working to turn Mila's treatment into a brand new path to treat children across hundreds of devastating genetic diseases.

 

 

GENE THERAPY

Together with renowned gene therapist Dr. Steven Gray, our foundation began the work on a new gene therapy direct to the brain for children with Mila’s type of Batten Disease (CLN7). This promising approach, which is still in the pipeline, would replace the broken gene with the corrected one in a one-time shot into the spinal fluid. Our trial is being developed by the University of Texas Southwestern in partnership with Aashi’s Hope Foundation and the Batten Hope foundation.

 

 

BASIC SCIENCE

In addition to funding therapies, our foundation is dedicated to better understanding the basic science behind Batten Disease. Currently, we support scientists trying to crack the CLN7 gene by hosting an annual Batten CLN7 symposium with researchers and physicians from around the world. We also provide grant funding to labs working on biomarker discovery and data collection, with the hope of contributing to clinical and research-based advances in the diagnosis and treatment of Batten as a whole.

 

 

Coming Soon:

2019 Batten Symposium


mILASEN - hOW IT WORKS

 
 

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.

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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.