Finding a Cure for Fragile X
Research aimed at finding Fragile X syndrome treatments is exploding. Why are so many pharmaceutical and biotech companies investing in this orphan indication? FRAXA chief scientific officer Dr. Michael Tranfaglia explains the many reasons Fragile X is such a hot topic.
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Dr. Mike Tranfaglia explains how FRAXA prioritizes research and the importance of looking at research from multiple angles. “It’s not either-or. It’s not we have a definitive treatment or we have a new drug treatment or we have a repurposing treatment. We can have all of those things, mixed or matched, in a personalized medicine kind of way and I think that’s what we’re headed for.”
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Dr. Mike Tranfaglia shares what FRAXA is excited about as we work through reviewing all of the submitted Fragile X research grant applications. We find it especially exciting that so many new clinical trials are starting right now, as our major emphasis is getting the drugs and other treatment strategies that we have tested in the Fragile X mouse model to patients in clinical trials.
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The Fragile X and Autism-Related Disorders Gordon Research Conference is a biannual event that provides an international forum for the presentation and discussion of frontier research in these conditions. Dr. Mike Tranfaglia explains why this is the premier conference for researchers and the scientific community.
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