Research study is open to participants worldwide to advance understanding and treatments for the rare disease Fanconi anemia.
What is gene therapy in FA, what do we know now, and what's next?
With a disease like Fanconi anemia, most of our attention is focused on physical health. Mental health is a component that may get overlooked, yet it is crucial to our overall wellbeing.
This summer, I had the honor of working in Dr. Alan D’Andrea’s lab at the Dana-Farber Cancer Institute in Boston. The people who research our disease are just as amazing as the people who have it.
Research study is open to participants worldwide to advance understanding and treatments for the rare disease Fanconi anemia.
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.
To better understand other issues facing the adult FA population, FARF formed an advisory council of adults living with FA. This group will provide input as FARF plans activities and policies.