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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. The bone marrow produces all blood cells: red blood cells, white blood cells and platelets. Red blood cells contain the protein hemoglobin that carries oxygen from the lungs to tissues. White blood cells fight infection. Platelets are important for blood to clot.
Children and young adults with dyskeratosis congenita are treated at Dana-Farber/Boston Children's through our Bone Marrow Failure 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 in-depth information on dyskeratosis congenita on the Dana-Farber/Boston Children's website, including:
We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”