Fanconi Anemia
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Stanford antibody regimen enables stem cell transplants without radiation or busulfan in Fanconi anemia
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A Stanford Medicine team reports that a single dose of the anti‑CD117 antibody briquilimab allowed three children with Fanconi anemia to undergo stem cell transplants without radiation or busulfan, achieving near‑complete donor cell engraftment in a phase 1b study published in Nature Medicine.