Targeted Research and Exploration Advancing Trial Models, Editing, and Next-generation Therapies (TREATMENT) Program at Boston Children’s

At Boston Children’s Hospital, we’re redefining what’s possible for children with rare genetic diseases.

More than 100 million children worldwide live with rare genetic diseases. Many remain undiagnosed, and few have effective treatments.

The TREATMENT Program unites families, researchers, and partners to accelerate breakthroughs in rare disease diagnosis and therapy.

Together, we’re building a future where every child receives the right diagnosis and access to life-changing care.