Explore the connections across the drug discovery and development continuum- linking patients with their life-saving drugs.
Mila was diagnosed in January 2017 with Batten disease; a rare disease caused by genetic mutations that leads fat to accumulate in nerves and brain cells. Her type of Batten's is particularly rare and fatal with no therapy available. Her family is on a mission to turn her incredible story into a new treatment path for children across hundreds of devastating diseases.
others in the Batten's genome database share Mila's same mutation
chance of her brother developing the disease
Mila received her first dose of Milasen at Boston Children's Hospital on January 31, 2018, after the FDA reviewed all the data and approved Mila's trial within a week. Along with the clinical team at Boston Children's Hospital, we achieved for Mila what had never been done before: a novel drug treatment within a year of her diagnosis, and approved treatment for her just six weeks after the first trial.
year to a novel drug treatment
weeks to approval from first trial
Boston Children's Hospital is ranked the number one pediatric hospital in the nation by U.S. News and World Report. It is home to the world's largest pediatric research enterprise, the leading recipient of pediatric research funding from the National Institutes of Health, and the primary pediatric teaching hospital for Harvard Medical School. Boston Children’s treats more children with rare diseases and complex conditions than any other hospital.
beds
specialized programs
pediatric hospital in the nation by U.S. News and World Report
Charles River has several decades of IND research experience, a unique range of services, and best-in-class expertise to help clients successfully initiate and complete an IND-enabling program on time and within budget. Of course, the same experience can also be applied to the design of suitable studies or programs for submission to any global regulatory authorities. We were proud to support Dr. Yu with regulatory consulting, toxicology and behavioral testing, bioanalysis, and drug product safety testing.
scientific advisors and growing
years of cumulative experience
years average experience per team member
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.
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Mila’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.
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Learn how antisense oligonucleotides are moving from obscurity to a viable drug strategy.
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Mila fought hard against Batten disease, a rare fatal condition with no cure. Her story is now giving new hope to millions with genetic disease.
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Mila’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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