At Boston Children’s Hospital, we’re redefining what’s possible for children with rare genetic diseases.
Families from around the world turn to Boston Children’s for answers they can’t find elsewhere.
Through the TREATMENT Program, families can join research studies, share data, and connect with experts focused on compassionate, coordinated care.
The TREATMENT Program: Hope in Action
Our Targeted Research and Exploration Advancing Trial Models, Editing, and Next-generation Therapies (TREATMENT) Program brings together physicians, scientists, and families to accelerate progress in understanding and treating rare genetic conditions.
With the TREATMENT Program, we’re:
- Improving care today with precise diagnoses, personalized support, and ongoing monitoring
- Advancing cures for tomorrow by developing and testing innovative therapies — including gene therapy, RNA-based treatments, and more
- Creating a global community of patients and families who power our research by sharing their experiences, stories, and data
How Families Help Shape the Future
We depend on the involvement of families like yours. Here’s how you can contribute:
Join Our Research Community
- Help researchers understand how rare conditions change over time
- Participate in online surveys or contribute medical records securely
- Provide samples (blood, urine, or skin cells) to help us model disease and test new therapies
Help Improve Diagnosis and Care
- Support early detection through newborn screening advocacy
- Inform diagnostic labs to include your child’s condition in testing panels
- Join natural history studies to guide future treatment strategies
Power Treatment Innovation
- Participate in studies testing safety and effectiveness of new therapies
- Help us understand the right “treatment window” when therapies are most likely to help
- Contribute to drug repurposing efforts, gene editing research, and more