Researchers report promising outcomes from clinical trial in patients with dyskeratosis congenita

Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center report promising outcomes from a clinical trial with patients with a rare form of bone marrow failure who received a hematopoietic stem cell transplant after pre-treatment with immunosuppressive drugs only. This is the first trial reporting successful transplant in dyskeratosis congenita patients without the use of any radiation or conventional cytotoxic chemotherapy beforehand.

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