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 truly personal medicines which Mila’s story has opened up, and making it accessible to many more across hundreds of rare diseases. Our hope is that our work will give Mila and many others like her a second chance at life.

Here’s how we are accomplishing our mission…

 

ASO TRIAL

Our foundation started an exciting new Antisense Oligonucleotide (ASO) treatment which Mila is currently undergoing. Led by the pioneering scientist/neurologist at Boston Children’s Hospital, Dr. Timothy Yu, this first-ever truly personal medicine, called Milasen, targets Mila’s disease-causing mutation. We are now working to turn Mila's treatment into an entirely new path for hundreds of rare diseases.

New England Journal of Medicine

New York Times


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 will replace the broken gene with the correct one in a one-time shot into the spinal fluid. The trial is being developed by the Univ. of Texas Southwestern in partnership with Aashi’s Hope and Batten Hope.

New York Post

Today Show


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 the disease, the more effective treatments will be.

 

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.


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 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.