Research aimed at finding Fragile X syndrome treatments is exploding. Why are so many companies investing? FRAXA CSO Dr. Michael Tranfaglia explains many reasons Fragile X is a hot topic.
This team is defining Fragile X “molecular signatures” to use as biomarkers. They’ll test CBD and other drugs in mice and compare findings to human brain data.
Depression is common in Fragile X, but current antidepressants need FMRP to work. Researchers will screen FDA-approved drugs to find effective options for FXS.
The FMR1 gene is renamed to “Fragile X Messenger Ribonucleoprotein 1” gene. The Fragile X protein, FMRP, is "Fragile X Messenger Ribonucleoprotein." "Mental retardation" is not used.
Dr. Mike Tranfaglia explains how FRAXA prioritizes research. "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.
Behavioral problems such as hyperactivity and aggression are more frequent in children with Fragile X syndrome, becoming less common as they grow through adolescence into adulthood.
Many new clinical trials are starting, with existing and investigational drugs and other treatment strategies that have been tested in the Fragile X mouse models moving to patients in clinical trials.
The Fragile X and Autism-Related Disorders Gordon Research Conference is a biannual event, an international forum for the presentation and discussion of frontier research in these conditions.
Archie really loves fire engines and trains as well as bells. Archie has had a few struggles this past year as his mom was diagnosed with cancer but thankfully she’s now cancer free.
Enhancing PKCε in early development normalized oxytocin, AMPAR signaling, and adult behavior in Fragile X mice, highlighting PKCε as a promising therapeutic target.
Dr. Peter Kind and Dr. Nahum Sonenberg share their optimism about the next 10 years of Fragile X research. They discuss where they think the next big discoveries will emerge.
FRAXA funded the LovaMiX trial of lovastatin + minocycline for Fragile X. 2022 results show safety and support continued study of this dual-target treatment approach.
FRAXA president and co-founder, Katie Clapp, and and FRAXA Scientific Advisor, Dr. Elizabeth Berry-Kravis, presented at the Neurodevelopmental Drug Development Summit.
Fragile X syndrome might be treated by reactivating the gene which is shut down in the syndrome. Researchers were able to reduce FXS symptoms by inserting the FMR1 gene into the brains of young mice.
Jonathan has changed a lot as he’s gotten older. He’s become calmer and less aggressive and has developed into a very social man! He still struggles with anxiety.
FRAXA’s Dr. Mike Tranfaglia talks with Autism Science Foundation’s Allison Singer about collaborations between the Fragile X community and other disability organizations as we look to the next 10 years.
