Milas Miracle Foundation Inc.

“Expenses administration expenses:marke 1,643 administration expenses:marke 1,211 administration expenses:marke 148 administration expenses:gener 833 travel expenses - pgm:airfare 4,594 travel expenses - pgm:airfare 24 travel expenses - pgm:ground 1,156 travel expenses - pgm:lodging 8,076 travel expenses - pgm:meals - 5,022 travel expenses - pgm:other t 8,654 travel expenses - pgm:parking 852 travel expenses - pgm:parking 96 travel expenses - pgm:rail/tr 131 travel expenses - pgm:tempora 11 administration expenses:gener 1,024 bank fees 316 dues & subscriptions 9,012 miscellaneous 13 taxes & licenses 3,091 total 45,907”

“The mission of mila's miracle foundation is to find and fund paths to a cure for batten and other life-threatening neurological disorders. Families in this community have been pivotal in raising the necessary funds and awareness. Together, with our incredible scientists and partner organizations, we hope that our work will improve the prognosis of batten disease and other neurodegenerative diseases. Through our fundraising - which includes grassroots efforts, individuals and family foundations, and grants - our foundation work has focused on the following program initiatives to date: the expansion of individualized medicines; clinical trials & programs, including an antisense oligonucleotide (aso) clinical trial, a cln7 gene therapy trial, and a batten & neurodegenerative disease clinic; and basic science research for batten disease, including a pediatric cell atlas of batten pathobiology and therapeutic response.”

“Expansion of individualized medicines - at present, the foundation is focused on helping to grow the new field of individualized medicines, which mila's story first opened up. Alongside mila's doctor (dr. Timothy yu) and his team at boston children's hospital, we are working collaboratively with leaders in the field to rethink the drug development process so we can safely and rapidly scale n-of-1 treatments and address the global health crisis of rare genetic diseases. Our "mila to millions" initiative is prioritizing two key areas to successfully bridge the gap between the need for treatments, the existing technology to develop them, and the missing infrastructure to expand access to them. Our strategic pilots in the uk and us are aimed at providing new models of access to individualized medicines and our impact campaign is aimed at shifting the public mindset to influence the critical decisions needed to pave this pathway forward. Our hope is to go from mila to millions, making individualized medicines accessible to many more children across thousands of rare diseases.”

“Clinical trials & programs - to date, the foundation has funded two (2) clinical trials and one (1) clinical program for children with batten disease: 1.) an antisense oligonucleotide (aso) clinical trial - the foundation partnered with boston children's hospital to fund, develop, and administer milasen, the first-ever individualized medicine designed for one person. Led by the pioneering scientist/neurologist, dr. Timothy yu, this aso treatment, which mila received for three years, targeted her individual disease-causing mutation and proved that novel treatments similar to this could be developed and deployed to treat children affected by a number of other rare genetic disorders. 2.) a cln7 gene therapy trial - together, with renowned gene therapist dr. Steven gray, the foundation began the work on a novel gene therapy direct to the brain for children with mila's variant of batten disease, cln7. This promising approach replaces the broken gene with he 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. 3.) neurodegenerative disease clinic - the foundation helped fund and launch a batten & neurological disease clinic at children's hospital colorado in 2020. The purpose of establishing this center of excellence was to provide families with children affected by neurodegenerative diseases a place to receive specialized multidisciplinary care all in one visit during a single appointment conducted annually and/or semi-annually, based on the child's personalized care plan. Simultaneously, research data is collected to help better understand these diseases and eventually inform future treatment paths.”

“Basic science research - to date, the foundation has funded three (3) basic science research projects for batten disease in the us and europe. By supporting 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, we are learning more about this devastating childhood condition with the hope of improving future treatment options. Early research projects funded by the foundation have led to findings and results published in the journal of clinical investigation, which demonstrate that an "aav9/mfsd8 gene therapy is effective in pre-clinical models of neuronal ceroid lipofuscinosis type 7 disease". Additionally, donated tissue samples, funding, and patient-partnered research support provided by the foundation led to the creation of the first-ever pediatric cell atlas of batten pathobiology and therapeutic response.”