Stem Cell Transplant Successfully Treats Bone Marrow Porphyrias

Stem Cell Transplant Successfully Treats Bone Marrow Porphyrias

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Stem Cell Transplant Successfully Treats Bone Marrow Porphyrias

A blood stem cell transplant can successfully correct defects in the body’s ability to make heme — a molecule that helps transport oxygen through the bloodstream — in people with bone marrow-based porphyrias, a small study showed. Thus, in patients with severe porphyrias, stem cell transplants should be considered early on in the treatment plan to minimize organ damage, the researchers said. The study, “Hematopoietic stem cell transplant for erythropoietic porphyrias in pediatric patients,” was…

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