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I’d like to tell you about a journey that changed my life forever. FA has taught me a couple of life lessons that you would expect to hear from a man late in his 90s.
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Learn more about Dr. Paula Rio, who works on gene therapy and gene editing in FA
In early October, the U.S. Food and Drug Administration awarded 12 new clinical trial research grants to advance treatments for rare diseases. One of the trials is a phase 2 study of quercetin chemopr
At the FAdult Meeting in Chicago, Mary-Beth (a blogger & adult with FA), took over FARF's Facebook for a day and brought viewers behind the scenes at the meeting.
What is gene therapy in FA, what do we know now, and what's next?
FA organizations in Europe are joining together to form a unified European Group.