The research enterprise at Boston Children’s Hospital, comprising more than 3,000 researchers, is the world’s largest at a pediatric center. Our work is fueled by a deep understanding of disease coupled with world-class discovery platforms, including genetics and genomics, gene editing, bioinformatics, proteomics, bioengineering, image analysis, multiple animal models, biobanks and disease-specific stem cell lines. We have special expertise in rare disease discovery and see large, highly diverse patient populations.
$388 million in total research funding FY2018 — #1 NIH funding for pediatric research
Dr. Yu and his team of scientists pulled off something unprecedented, something seemingly impossible. In the space of 10 months, they pinpointed Mila’s rare mutation, designed a drug to bypass it, engaged industry partners and FDA regulators, and launched a treatment trial — just for Mila.
Early encouraging results are reported in The New England Journal of Medicine. They could open a new path, showing it’s possible to create personalized genetic therapies for children with rare conditions — and to do so quickly.
© 2005-2019 All Rights Reserved