Fragile X Research Q&A with Dr. Berry-Kravis on RECONNECT results, placebo effects, outcome measures, methylation, and research directions for families.
Join the Schutz family in their journey with Fragile X syndrome. Discover Wally’s inspiring progress, the impact of clinical research, and how donations to FRAXA fuel hope for breakthroughs to support families affected by Fragile X.
This project will examine how CBD and other drugs targeting the endocannabinoid system affect hyperexcitable Fragile X neurons to identify new treatment strategies.
Disappointing results have been published from Zynerba Pharmaceuticals’s phase 3 clinical trial of Zygel™. 212 children and adolescents 3 to 17 years were given Zygel or placebo for 12 weeks.
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.
Each year, FRAXA funds a diverse portfolio of research. FRAXA Fellowships and Grants are seed funding for the future, the feedstock for the Fragile X treatment development pathway.
Why does obesity challenge so many people with Fragile X? Dr. Caku’s team has found that Fragile X syndrome causes changes in the tiny organisms that live in our gut.
Zynerba Pharmaceuticals received advice from the U.S. Food and Drug Administration (FDA) on the design of a Phase 3 clinical trial of Zygel as a cannabidiol treatment for Fragile X syndrome.
This study tested CBD (cannabidiol) treatment in male and female Fragile X mice to learn how and why it works and whether gender affects responses to CDB treatment.
The team studied how inhibitory brain circuits malfunction in Fragile X and tested ways to restore balance by targeting mGluR and endocannabinoid signaling.
Zynerba presented clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel, may reduce behavioral abnormalities in children with Fragile X syndrome who have more severe disease.
Administering a cholesterol drug alongside an antibiotic eases atypical behavior and restores the signaling balance in the brains of people with fragile X syndrome.
This FRAXA grant studied why people with Fragile X are overly sensitive to sound and tested drug strategies to calm the brain’s overactive auditory circuits.
FRAXA funds biomarker research to identify objective, measurable signals of Fragile X, helping predict who will respond to treatments and improving clinical trial success.
Fragile X disrupts endocannabinoid signaling. This study in mice demonstrated that correcting it may calm brain hyperexcitability and improve symptoms.
CB1 blockade with rimonabant reversed cognitive, sensory, and seizure symptoms in FXS mice, highlighting the endocannabinoid system as a therapeutic target.
FRAXA-funded studies found faulty endocannabinoid signaling in Fragile X brain circuits for reward and emotion, and boosting 2-AG restored normal function.
