Research at Boston Children's


Patient-driven Science

The first repair of a congenital heart defect, the first remission of leukemia and a groundbreaking treatment for sickle cell disease: These are just a few of the many research “firsts” we’ve achieved over the past 150 years to save lives. And we are far from finished.

An icon of a medical cross inside of a heart

Our commitment to cures

We'll never stop looking for answers to childhood illnesses.

An icon of a medical cross inside of a heart

Our commitment to cures

We'll never stop looking for answers to childhood illnesses.

Precision Medicine

Doctors have long wondered why children with the same diagnosis respond differently to treatment. The answer is in their genes. Our researchers are delving into the genetic underpinning of diseases and developing state-of-the-art methods to screen for these conditions.

For example, more than 300 genes linked to epilepsy have been discovered by our Epilepsy Genetics Program researchers and their colleagues worldwide. By pinpointing the causes, we can ultimately pinpoint the treatments for the 1 in 26 people affected by this complex condition.

Our experts are finding chemical markers in the body that could be used to identify disorders, such as endometriosis, transplant rejection and concussions. 

And our scientists are examining how the body’s microbes can affect disease symptoms. For example, we’re testing a cutting-edge treatment for food allergies based on rebalancing bacteria in the gut.

Our Research Impact
3,000+
researchers and scientists, the world’s largest pediatric research enterprise
300
active clinical trials bringing hope to kids and families, with roughly 1,700 patients enrolled
1,700+
patents issued for Boston Children's discoveries
90%
of our patented discoveries help adults as well as children

Data Revolution

Boston Children’s expertise doesn't end in the lab or by the bedside. We’re at the forefront of data analytics and predictive technologies. Recent innovations include the world’s first artificial intelligence platform to predict suicide risk in young people and a brain-based tool to screen infants for autism. Our experts are identifying complex patterns from patient data and helping clinicians everywhere make informed decisions for the kids in their care.

Discoveries Benefit Adults, Too

Unlike adults, children are not subject to lifestyle behaviors like smoking or drinking alcohol that can contribute to disease, so our experts can more clearly see the root of their illnesses. And because many conditions begin in childhood, Boston Children’s discoveries pave the path to cures for adults as well as children.

A Lifesaving Solution

A new surgery healed Presley's heart–and will help other kids, too.

A Lifesaving Solution

A new surgery healed Presley's heart–and will help other kids, too.

A History of Breakthroughs

Across the 150-year history of Boston Children’s, our researchers and scientists have led the way in finding new treatments and cures for childhood illness. A few of our many “firsts”: 

  • 1948: Achieved the first remission of leukemia
  • 1949: Developed the first polio vaccine
  • 1959: Made a discovery to save premature babies from severe lung problems
  • 1963: Developed the first measles vaccine
  • 1983: Developed an effective treatment for sickle cell disease
  • 1984: Performed the first liver transplant in an infant
  • 2001: Performed the first successful repair of a fetal heart defect
  • 2010: Treated the first patient with a new, lifesaving therapy for “bubble boy disease,” a rare genetic disorder
  • 2017: Built an AI system predicting severe mental health conditions years in advance 
  • 2021: Designed the first prosthetic heart valve specifically for kids
  • 2022: Developed a gene therapy to cure a rare neurodegenerative condition in children
  • 2023: Designed and performed a groundbreaking brain surgery on a baby in the womb 

Today

We’re continuing to pursue life-changing discoveries, including creating new drug-delivery methods, restoring hearing in children, and developing a new therapy to attack neuroblastoma, an often-deadly cancer.

With your generous support, we can save even more kids.

With Your Support, We Find Answers

Help us create new treatments and cures that save kids’ lives.

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