Explore the connections across the drug discovery and development continuum- linking patients with their life-saving drugs.
Find out more about Mila’s story and personalized medicine development.
Mila's Mom, Julia Vitarello joins our Vital Science podcast to tell the story of Mila’s diagnosis and her incredible journey since; from diagnosis to Mila’s experimental genetic treatment, created just for her.
Learn MoreMila’s groundbreaking treatment, the first created and approved for a single patient, has given hope to thousands of rare-disease patients around the world. Hear Julia Vitarello, Mila’s Mom, and Dr. Timothy Yu discuss their plans to help other children with rare disease.
Learn MoreLearn how antisense oligonucleotides are moving from obscurity to a viable drug strategy.
Learn MoreMila fought hard against Batten disease, a rare fatal condition with no cure. Her story is now giving new hope to millions with genetic disease.
Donate NowMila’s mother, Julia Vitarello, and other scientific leaders from around the world have launched the N=1 Collaborative; turning Mila’s story into a movement to help solve the global health crisis of rare genetic diseases.
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