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.
Company Details
- Employees
- 2
- Founded
- -
- Address
- Longmont, Co 80503, Us
- Industry
- Non-Profit Organizations
- Website
- https://www.milasmiracle.org/
- Keywords
- Mercer Island.
- HQ
- Longmont, CO
Mila's Miracle Foundation Questions
Mila'S Miracle Foundation's website is https://www.milasmiracle.org/
Mila'S Miracle Foundation's LinkedIn profile is https://www.linkedin.com/company/milas-miracle-foundation
Mila'S Miracle Foundation has
2 employees.
View email and phone details for 2
employees at Mila'S Miracle Foundation.
Mila'S Miracle Foundation's industry is
Non-profit Organizations
Mila'S Miracle Foundation's top competitors are
Charley's Fund,
Genomics England,
Everyone Medicines,
Gabaa Receptor Alliance,
Cure Gm1 Foundation,
N=1 Collaborative,
Arbor Biotechnologies,
Liberate Bio,
Institute For Healthcare Improvement / National Patient Safety Foundation,
Fam177a1 Research Fund.
Mila'S Miracle Foundation's categories are Non-profit Organizations
Mila'S Miracle Foundation's founding year is 2016
