FA researchers in Spain have published results from their gene therapy trial, showing that gene-corrected hematopoietic stem cells engrafted in the bone marrow of four people with FA.
Publication spotlight on Fanconi anemia from September 2019's Blood Review scientific journal
Well before her diagnosis of FA, our daughter was a model in resiliency for us. When she was finally born, we called her Mighty Mouse. Small, but resolute. Our model of resilience. Our Norah.