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We first learned something was wrong in 2004. I was a normal, healthy kid until I went to my pediatrician for my 13-year-old booster shot and blood draw. That was the first sign something was off.
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To fast-track therapies for people with Fanconi anemia (FA), it’s often best to start by examining progress that’s already been made in the general population.
La Anemia de Fanconi se consideraba una enfermedad huérfana por lo poco frecuente y lo poco que se conocían sus causas, sus conexiones a otras enfermedades, y sobre la existencia de tratamientos.
An update on the international Fanconi anemia gene therapy trial
An update for families about the FA pathway and fertility
People with FA have a 500- to 700-fold increased risk of developing head and neck tumors when compared to the general population. Alternative therapeutic options are desperately needed.