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.
This team is designing tiny “peptidomimetic” drugs that mimic FMRP’s function to rebalance protein production in the brain, aiming to treat Fragile X at its source.
Thirteen centers across the US enrolled young children with Fragile X in a large-scale clinical trial of Novartis AFQ056, an mGluR5 blocker for Fragile X which failed in adult human trials.
FRAXA-funded open-label trial found that metformin led to increased GABA-mediated cortical inhibition, suggesting metformin modulates core Fragile X pathways.
Healx has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for a Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).
British startup Healx has secured FDA approval for a phase 2a clinical trial of an AI-discovered compound that could help manage the symptoms of the genetic disorder Fragile X syndrome. The start of the trial marks another milestone in the use of artificial intelligence to help find new applications for existing drugs by mining patient records and research databases.
FRAXA Investigator Dr. Sumatra Chattarji investigated the synaptic basis of deficient conditioned fear and its reversal in Fragile X syndrome rats. Results published.
AI and FRAXA-DVI identified a drug + supplement combo that reversed all Fragile X symptoms in mice. A clinical trial tested this in adults with Fragile X.
