FRAXA Announces 2012 Fragile X Research Awards

April 3, 2012

2012 Fragile X Research Awards Total $1,132,923

$1,132,923 in New Program Grants, Postdoctoral Fellowships, and Renewals have been approved for the next year. We are funding over $846,000 in new projects; renewals total $285,678 and will increase as additional projects reach their one year mark.

Functional Interplay Between FMRP and CDK5 Signaling

September 13, 2014

FRAXA-funded work showed CDK5 signaling is disrupted in Fragile X. CDK5 drugs are in development for Alzheimer’s so this pathway offers a promising new FX treatment angle.

Small Molecules To Target r(CGG) Expansions to Treat Fragile X Syndrome

January 24, 2014

FRAXA-funded scientists created small molecules that target the CGG repeat “off-switch” in Fragile X, aiming to restore the missing FMRP protein at its source.

Potassium Channel Modulators to Treat Fragile X

January 7, 2014

FRAXA-backed Yale discoveries tied Fragile X to Kv3.1/Slack channel defects—leading to a partnership with Autifony to develop targeted treatments.

Social Behavior as an Outcome Measure for Fragile X Clinical Trials

September 13, 2013

FRAXA funding helped identify reliable social behavior tests in Fragile X mice and showed an mGluR5 treatment could improve sociability, guiding future trials.

Translation-Independent Functions of FMRP in Excitability, Synaptic Transmission and Plasticity

September 12, 2013

Study pinpointed presynaptic calcium dysfunction as the driver of STP defects in Fragile X, and BK channel activation restored normal synaptic signaling.

Glycogen Synthase Kinase-3 (GSK3), Lithium and Fragile X

September 12, 2013

Dr. Jope found that lithium (at usual therapeutic doses) and investigational GSK3 inhibitors can reverse a number of cognitive deficits in FMR1 knockout mice.

The mTOR Pathway in Fragile X Syndrome

September 6, 2013

FRAXA-funded research showed that blocking S6K1 in Fragile X mice improves social, behavioral, and physical symptoms.

Matrix Metalloproteinase Therapeutic Treatments for Fragile X Syndrome

September 2, 2013

Dr. Broadie showed that MMP enzymes disrupt synapse development in Fragile X. MMP inhibitors (e.g. minocycline) improved connectivity and behavior in fruit flies.