FROM MILA TO MILLIONS
Genetic disease is a global health crisis affecting hundreds of millions of people worldwide. Of these, tens of millions are children who will die before the age of five. There are thousands of individual genetic diseases, most of which are rare. Together, these rare diseases impact more people than cancer and AIDS combined, yet few to no treatments are available. We’re on a mission to change this.
Mila’s story showed what is possible: that a medicine can be made to target the underlying genetic cause of disease, often a single mutation, even if unique to just one person. Now, Mila’s treatment is setting the stage for a new paradigm of individualized medicines — one that could offer a unique approach to solving the global health crisis of rare genetic disease.
OUR vision
Instead of one drug for tens of thousands of people, Mila's story lights the way towards a future of tens of thousands of drugs each for one or very few. We have the science and technology, we have millions of dying children who could benefit from these treatments, but there is no rational framework for making this approach accessible to those who need it urgently.
So how do we go from Mila to Millions?
We need to rethink our current system from the bottom up. Together, with industry experts, regulators and academia alike, we’re working strategically to lay the groundwork for a new regulatory path that will address this unique approach to treating patients.
OUR impact
Milasen gained global attention for being the first medicine designed for one person. Now, we’re determined to make similar treatments possible for many more children with rare genetic diseases. Speaking at presentations and meetings worldwide, we’re creating awareness and opportunity for the future of individualized medicines to lead the way forward… from Mila to Millions.
Genomics England Discovery Forum 2022
MORE PRESENTATIONS
RE(ACT) Congress & IRDiRC Conference 2023
Harvard Medical School Boehringer Ingelheim Executive Education Webinar 2023
Oxford-Harrington Rare Disease Centre Rare Disease Day Webinar 2023
Danaher Leadership Conference 2023
University of Oxford Department of Paediatrics IDRM Seminar 2022
MIT Sloan School of Management Healthcare Finance Seminar 2022
Ultragenyx Leadership Summit 2022
RNA Therapeutics Symposium 2022
ASGCT Annual Meeting 2022
ASCPT Annual Meeting 2022
N=1 Collaborative End-of-Year Meeting 2021
UCSF-Stanford CERSI-FDA Workshop on Prenatal Somatic Cell Gene Therapies 2021
CZI n-of-1 Meeting Series 2021
NYU Langone Health Pediatric Gene Therapy & Medical Ethics Working Group 2021
Colorado BioScience Association Briefing 2020
Children’s Hospital Colorado’s NSI Burns Night 2020
The Precision Medicine World Conference (PMWC) 2020
FDA Meeting: Promoting Effective Drug Development Programs 2019
Stanford Medicine Pediatric Grand Rounds 2019
GoldLab Symposium 2019
FDA’s Critical Path Innovation Meeting 2019
MIT’s Broad Institute Rare Disease Day 2019
Foundation for Angelman Syndrome Therapeutics Gala 2018
Boston Children’s Hospital’s Manton Center for Orphan Disease Research 10th Anniversary 2018
Undiagnosed Disease Network UDN Steering Committee 2018
American Society of Human Genetics Conference 2018
AWARDS
BIO: Women’s History Month 2021 (28 pioneering women in science and biotechnology to know)
DISORDER: The Rare Disease Film Festival Film Selection 2020
NORD Rare Impact Award Honoree 2020
If you’d like Julia to speak at your upcoming event, please contact us using the form below.
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.