A rare, genetic disorder that prevents bone marrow from producing healthy blood cells and platelets, often associated with birth defects, blood disorders, and cancers in children and adolescents, has ...
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Why metabolism matters in Fanconi anemia: How a rare genetic disorder disrupts energy pathways
Experts at Cincinnati Children's have uncovered striking metabolic differences in people with Fanconi anemia (FA), a rare genetic disorder that causes bone marrow failure and dramatically increases ...
An international study led by researchers from Children's Hospital of Philadelphia (CHOP) has discovered an important biological cause of Fanconi anemia, a rare inherited disorder that almost ...
Gene therapies could have a major impact, although the cost and complexities could inhibit their wide rollout, study authors said. New advancements in gene therapy and hematopoietic stem cell ...
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