Boston Children's.
Where the world
comes for answersBut where do those answers come from?But where do those answers come from?
We're launching a global initiative to transform children's health.
We need visionary partners, like you, to join us.
How do we wipe out childhood disease?

Cure the Incurable
Over 150 million children suffer from rare diseases with no cure; nearly a third will die before age 5. We’re changing that with new targeted therapies.

Predict the Unpredictable
Generations will be shaped by what we cannot yet see. We’re readying systems and people to take on the world’s next big challenges.

Lead the Way
Boston Children’s has transformed children’s health worldwide. Now we’re shaping the future of pediatric medicine.
Discoveries
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In the News
Helen's Story
Helen Obando is the youngest person to ever participate in a clinical trial for sickle cell anemia. The results have been life-changing. Is she cured?
How do we wipe out childhood disease?
Cure the Incurable
Over 150 million children suffer from rare diseases with no cure; nearly a third will die before age 5. We’re changing that with new targeted therapies.
Predict the unpredictable
Generations will be shaped by what we cannot yet see. We’re readying systems and people to take on the world’s next big challenges.
Lead the Way
Boston Children’s has transformed children’s health worldwide. Now we’re shaping the future of pediatric medicine.
Firsts that changed the world
Boston Children’s stunning breakthroughs have saved millions of lives.
With your partnership, we can do so much more. See a sample of our firsts below:

1948
The first remission of leukemia
Sidney Farber, MD, was the chief of pathology at Boston Children's at a time when a leukemia diagnosis was a death sentence. But his hunch that antifolate could potentially be a cure for acute lymphoblastic leukemia led to the first clinical trials to show that a drug could treat cancer. Farber's work helped establish the field of chemotherapy, and brought us a future where 80 to 90% percent of children with leukemia are cured.

1949
The first polio vaccine
Thomas Weller, MD, and his boss, the great virologist, John Enders, PhD, were experimenting with chicken pox when Weller added poliovirus to a few flasks of leftover culture medium. Never has an afterthought done more for humanity. The poiliovirus poliovirus grew, opening the door for the development of polio vaccines. The discovery earned Weller, Enders and their colleague Frederick Robbins, MD, the Nobel Prize, and led to an explosion of vaccine development.

1959
Saving premature babies
Mary Ellen Avery, MD, realized that preemies' immature lungs lacked surfactant, a foamy material that lowers the surface tension of the lungs' air sacs, enabling them to inflate again after exhaling. She helped identify the components of surfactant, the cells that produce it and when it develops in the fetal lung. She also inspired the research that turned her discovery into a treatment. Millions of premature babies now thrive, thanks to Avery.

1963
The first measles vaccine
John Enders, PhD, tasked a young associate with isolating the virus that causes measles. For weeks, Thomas Peebles collected blood samples from sick students at a private boys' school near Boston. He told each, "Young man, you are standing on the frontiers of science." The strain of measles Peebles ultimately isolated is named for the boy whose blood provided the virus, David Edmonston. It is still used in measles vaccines worldwide.

1983
The first effective sickle cell treatment
As a kid, Claudia De Pass was spending more time in the hospital than at school due to sickle cell disease. But then she became the first sickle cell patient to take hydroxyurea, a blood cancer drug advocated by Boston Children's Chief of Hematology and Oncology, David Nathan, MD. The drug changed Claudia's life—and lives of patients around the world. Today, David Williams, MD, is leading clinical trials where gene therapy may cure sickle cell disease for good.

2001
The first fetal correction for HLHS
Hypoplastic left heart syndrome (HLHS) leaves infants with one pumping chamber and a lifetime of limitations. So when Jennifer and Henry's baby was diagnosed prenatally with that condition, they contacted Wayne Tworetzky, MD, director of the Fetal Cardiology Program. Tworetzky proposed a daring solution: Correct the defect in utero. Jack was born with a healthy heart. His was the first successful fetal correction of HLHS. He now surfs and plays baseball, just like any other teenager.

Today
We are finding breakthroughs for sickle cell, rare disease, immune deficiency, cardiovascular disease, autism and thousands of other diseases and conditions, thanks to generous donors.
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1948
The first remission of leukemia
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1949
The first polio vaccine
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1959
Saving premature babies
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1963
The first measles vaccine
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1983
The first effective sickle cell treatment
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2001
The first fetal correction for HLHS
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Today
2 Nobel Laureates • 8 Lasker Award winners (America's Nobel) • 4 MacArthur "Genius Grant" Fellows • 27 HHMI Investigators/Alumni • More National Institutes of Health Grants than any pediatric institution

Partner with us
It's always there, on the horizon.
The next discovery, the next breakthrough, the next cure.
Talk with our team about how, together, we can transform the future of children's health.
Because the next big thing could be just the chance a child is waiting for.
Where the world comes for answers
Cure the Incurable
Precision medicine: Key to conquering “incurable” rare disease
Rare disease is not rare.
Worldwide, 350 million people suffer from rare diseases. Half are children. To put that in perspective, each year 400 thousand kids are diagnosed with pediatric cancer (a rare disease in and of itself).
How do we cure the incurable? Precision medicine.
As 80% of rare diseases are genetic, therapies must be personalized. Precision medicine techniques, like gene therapy, are very effective because they are tailored to the underlying cause. More support is needed to build on the promise of these technologies and make genomics part of routine health care. These investments can change the future for millions.
Ripple effect: Changing lives worldwide
Why have families from Argentina, Ireland and Vietnam travelled to Boston Children's for gene therapy? They count on our experts to deliver the lifesaving care their children need. They participate in a pioneering gene therapy clinical trial. They know we will never stop moving forward. Today, we’re accelerating discoveries with the potential to help countless children:
2010: We cured a severe immune disorder, "bubble boy disease," using gene therapy, and a new gene therapy approach for this disease is in trial.
2013: We launched a novel international gene therapy protocol for adrenoleukodystrophy (ALD).

2018: We started the first patient on a groundbreaking gene therapy trial for sickle cell disease, bringing us closer to a cure for children like Lamarcus (pictured). All patients in the trial are currently healthy and symptom free. Phase 2 of the study launched in 2022.
Today, our Gene Therapy Program is researching ways to make this expensive and complex treatment more accessible and enable more hospitals, particularly in resource-limited regions, to provide gene therapy.
Our International Precision Child Health Partnership with three other world leaders across three continents is tackling major child health challenges in what is believed to be the first major global collaboration of children's hospitals around genomics and child health.
Our Children's Rare Disease Cohorts Initiative has sequenced the DNA of nearly 10,000 patients with rare disease and their family members, uncovering novel mutations to develop new targeted therapies and connect patients with clinical trials.
Urgent needs remain
The National Institutes of Health currently funds just 6% of applications, and the field is only getting more competitive.
Philanthropic support would enable an incredibly robust research platform that can deliver:
- a critical mass of genetic data to identify the unknown drivers of disease.
- greater computing power for bioinformatics analysis and automated drug discovery.
- support to bring new medical devices to patients.
- stronger translational research initiatives and clinical trials to take new therapies across the finish line.
We have the plan, the team, the culture and the ideas to change lives worldwide. All we need is you.
Predict the unpredictable
Keys to predicting future events and saving lives
Worldwide, physicians, public health officials and patients are often "flying blind."
Every day, critical health decisions must be made without necessary information. With access to vital data, we can truly understand diseases and make well-informed decisions about how best to treat and prevent them.
Resources are scarce.

Worldwide, physicians, public health officials and patients are often "flying blind."
Every day, critical health decisions must be made without necessary information. With access to vital data, we can truly understand diseases and make well-informed decisions about how best to treat and prevent them.
Resources are scarce.
COVID-19 has shown just how quickly hospitals reach capacity and resources become severely strained. Expanding the availability of providers and increasing training will enable health care systems to better handle surges and assist areas and families with the highest needs.
Another crisis will emerge.
Even as we struggle to address the devastating impact of today's pandemic, another looms on the horizon. Preparing is absolutely essential. Having the tools to predict health crises and the experts ready to respond means faster implementation of public health strategies.
Ripple effect: Changing lives worldwide
Why do the World Health Organization, the Centers for Disease Control and Prevention and countless others partner with Boston Children's? For more than a century, we’ve been solving public health crises—polio, measles, Ebola. What’s next? Boosting COVID-19 immunity in the elderly and developing a vaccine against opioid overdose.
Importantly, we're delivering vital expertise to:
- inform and guide response to pandemics, mental health crises, obesity, bullying and so many other major issues impacting children.
- expand access to critical care for children and families and health education for care providers worldwide.
- advocate to protect the health and well-being of children and families everywhere.
Our HealthMap automated platform collects, validates and cross-references data from around the globe. Key findings from HealthMap are informing decision makers, including the World Health Organization and Centers for Disease Control, in real-time.
Our Computational Health Informatics Program (CHIP) breaks information silos, enabling the secure sharing of critical clinical information between health care systems. CHIP transforms how doctors diagnose and treat patients and fuels insights and strategies for major health issues.
Created at Boston Children's and used in every country and territory worldwide, OPENPediatrics delivers open-access, evidence-based online training that is bridging the gaps in health education and improving care.
Urgent needs remain
COVID-19 has magnified problems in health care systems around the world.
We can weather today's crises, while preparing for the ones we will face in the future, by:
- strengthening biomedical informatics platforms, surveillance systems and artificial intelligence to efficiently respond to outbreaks of disease.
- supporting children who experience trauma, including loss of a parent from illness or violence. Nearly a quarter of the world's children live in conflict or disaster-stricken regions. Vital services can reduce vulnerability and improve resilience.
- delivering essential training and education for health care workers.
The steps we take today will determine tomorrow's outcomes. Join us.
Lead the way
For more than 150 years, Boston Children’s has relentlessly sought to improve health not for one child or a hundred but for all children. Today, we:
- operate the world’s largest research enterprise at a pediatric hospital, with 3,300 scientific staff.
- provide 264 specialized clinical programs, with approximately 1,700 patients enrolled in active clinical trials.
- care for patients in more than 1.2 million clinical visits each year.
- support families coming to Boston from more than 100 countries.
- run the largest and most competitive training program in pediatrics.
- publish more than any other children's hospital.
- work tirelessly to deliver answers for the toughest challenges in child health.

Ripple effect: Changing lives worldwide
Pioneering discoveries at Boston Children's impact children around the globe. Here are just a few examples of how we continue to lead the way in breakthroughs that transform medicine:
- Our experts discovered the cause of lung injury in premature babies, leading to treatment that has saved more than 800,000 lives worldwide. Today, we are taking bold steps to increase the chances of survival and the quality of life for many newborns through advanced prenatal diagnostic tools, groundbreaking surgical and medical treatments, and unparalleled care that extends through childhood.
- Our specialists launched and shaped the field of pediatric heart surgery, performing the first pediatric open-heart surgery, then the first successful cardiac intervention in the womb. Today, we are on the cusp of eliminating the need for repeat open-heart surgeries by providing artificial heart valves and other novel devices that expand as children age.
- Boston Children’s is a powerhouse for basic and translational neuroscience, tackling the most difficult challenges, such as:
- predicting autism earlier in infancy.
- promoting regeneration of nerves in the central nervous system.
- preventing the emergence of anxiety.
- restoring hearing in patients with genetic hearing loss.
#1 in the nation: What does that actually mean?
For nine years in a row, Boston Children's has been ranked the nation’s #1 hospital by U.S. News & World Report. This first place ranking stems from data on a host of measures, most importantly outcomes. Patients surviving and thriving: That is our differentiator, and it’s the heart and soul of our institution.
Scores also are determined by more than 15,000 pediatric specialists voting where they would send the sickest children in their specialty. At Boston Children’s, we are not just top-ranked in all 10 specialties; we also have been first in more of those areas than any other institution for nine years running.
And that’s why we are the destination for complex care for the world’s pediatric population.
#1 in National Institutes of Health funding: Why?
Our faculty, their research, our commitment.
Decades ago, Boston Children's made research a priority, recruiting more Howard Hughes Medical Institute investigators—the gold standard in basic science—than our peers combined. Today, we rigorously support a deep bench of star scientists with breadth, depth and breakthroughs unlike any other.
To eradicate childhood disease, however, we cannot rely on government funding alone. The NIH does not support the bold early science that drives life-changing discoveries. For that, children here and around the world need you.