Research at Boston Children's Hospital

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.

michael rogers and danielle peterse

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More than 3,000 researchers & scientific staff

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1 million square feet of research space — and growing

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$388 million in total research funding FY2018 — #1 NIH funding for pediatric research

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A new frontier: Inside a custom drug

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.

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Dr. Yu batten disease

Boston Children's Hospital Innovation

BCH Innovation News @BCH_Innovation 10/11/2019 6:00:01 PM
“I think that some of the most exciting parts in #science are when you try to do something new, when there isn't a…
BCH Innovation News @BCH_Innovation 10/11/2019 4:00:02 PM
Shooting for the moon: From genetic diagnosis to custom treatment in under one year: #nof1…
BCH Innovation News @BCH_Innovation 10/10/2019 8:00:01 PM
Mila has an extremely rare mutation, and a drug made just for her by @timyu and his team @BostonChildrens:…
BCH Innovation News @BCH_Innovation 10/10/2019 5:00:02 PM
“Make the most of your time with Mila,” doctors said. But that wasn’t good enough for Mila’s parents — or for…
BCH Innovation News @BCH_Innovation 10/9/2019 4:54:35 PM
RT @WBUR: A @BostonChildrens doctor who's a pediatric trauma surgeon writes for @cogwbur about the dangers of driving and texting: https://…
BCH Innovation News @BCH_Innovation 10/7/2019 8:00:00 PM
A new study has identified the particular helper T cells that cause MS, as well as a protein on their surface that…
Building on our extraordinary leadership in pediatric science at Boston Children’s, we translate our findings — and those of others — to fundamentally change how disease is treated and cured.

David A. Williams, MD
Senior Vice President and Chief Scientific Officer