Dyskeratosis Congenita (DC)

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Clinical Trials for Pediatric Patients

What Is Dyskeratosis Congenita?

Normally, bone marrow produces all of the blood cells your child's body needs, but sometimes, genetic defects can disrupt the marrow's ability to make these vital cells. Dyskeratosis congenita (DC) is a rare genetic disorder that affects multiple parts of the body, including the bone marrow's ability to make blood cells. 

Dyskeratosis Congenita Treatment at Dana-Farber/Boston Children's

Children and young adults with dyskeratosis congenita are treated by our team of experts at Dana-Farber/Boston Children's through our Bone Marrow Failure and Myelodysplastic Syndrome Program, recognized as one of the nation's best pediatric treatment and research programs for bone marrow failure and related conditions. Our patients have access to advanced treatments and diagnosis, including DNA mutation identification and ongoing clinical trials investigating new treatments. Dana-Farber/Boston Children's is also home to one of the largest and most experienced pediatric stem cell transplant centers in the world. Stem cell (bone marrow) transplant is currently the only cure for the blood defects of DC.

Find more in-depth information on dyskeratosis congenita on the Boston Children's Hospital website, including answers to:

  • What causes dyskeratosis congenita?
  • What are the symptoms of dyskeratosis congenita?
  • How is dyskeratosis congenita diagnosed?
  • How is dyskeratosis congenita treated?
  • What is the latest research on dyskeratosis congenita?