FRAXA-funded researchers have taken another promising step forward.
A study from MIT’s Picower Institute for Learning and Memory reveals a potential strategy to treat Fragile X syndrome by enhancing NMDA receptors — key players in brain communication and learning.
Over several years, Dr. Stephanie Barnes was awarded a FRAXA Postdoctoral Fellowship in the Edinburgh lab of Dr. Emily Osterweil, followed by a visiting fellowship at MIT with Dr. Mark Bear. Her focus was to explore how NMDA receptors could be adjusted to improve learning and reduce Fragile X symptoms. That early work laid the foundation for a peer-reviewed study published in Cell Reports this year, which highlights this signaling pathway as a promising therapeutic target.
Targeting NMDA Receptors to Treat Fragile X Syndrome
By boosting the activity of a specific NMDA receptor subunit in the brain , the researchers were able to normalize protein synthesis in mouse models of Fragile X. Mice were calmer, learned better, and had fewer signs of overstimulation.
MIT Researchers Explain the Science Behind the Discovery
In an engaging video produced by the Picower Institute, the team walks through the science behind their findings and why it matters for Fragile X. It’s a great way to appreciate what’s happening in the brain and understand how this research could lead to real-world impact.
FRAXA-Funded Research Teams Deliver Results
This discovery is a reminder that FRAXA’s approach — funding early-stage, high-potential research — continues to bear fruit. Drugs that selectively target certain NMDA receptors are also being studied for other conditions, offering a potential fast track toward human studies.
NMDA receptors are just one of many promising targets being explored by FRAXA-funded scientists around the world.
It’s also a reminder that meaningful discoveries don’t happen overnight. They happen because researchers keep asking new questions, and because the Fragile X community continues to show up and support the science.
Video courtesy of the MIT Picower Institute
