Fragile X Syndrome: In Pursuit of a Cure Webinar
Global webinar “Fragile X Syndrome: In Pursuit of a Cure” on July 22, 2021 commemorated World Fragile X Day. Over 5,000 registered from more than 50 countries.
Pharmacotherapeutic Effects of Cannabidiol (CBD) in Fragile X syndrome (FXS) and Autism Spectrum disorder (ASD)
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.
Auditory Dysfunction in Fragile X Syndrome in a Mouse Model of Fragile X
FRAXA-funded studies found Fragile X mice show altered auditory circuit function with delayed startle timing and reduced prepulse inhibition, mirroring human sound sensitivity.
Towards Understanding the Role of FMRP in Human Brain Development Using Brain Organoids
Dr. Zhexing Wen and Dr. Peng Jin at Emory University School of Medicine join us in this seminar to present Understanding the Role of FMRP in Human Brain Development Using Brain Organoids.
Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model
Long-term, but not short-term, treatment with bryostatin-1 arrested some behavioral and cognitive symptoms in a mouse model of Fragile X.
Allos Pharma Revives Arbaclofen After 7 Years: New Hope for Fragile X Syndrome
Allos Pharma Inc launches a new development program for arbaclofen to treat Fragile X syndrome. This experimental drug missed its primary endpoint in clinical trials.
Positive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor Zatolmilast from Tetra Therapeutics
Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome. The results do not depend on carving out a subset of patients or post hoc analysis.
Use of EEG as a Biomarker for Diagnosis and Outcomes in Neurodevelopmental Disorders
This webinar features Charles A. Nelson III, PhD, Professor at Harvard Medical School and Carol Wilkinson, MD, PhD, Instructor at Boston Children’s Hospital.
Genome-wide Screen for FMR1 Reactivation in Human FXS Neural Cells
This team aims to turn the FMR1 gene back on in Fragile X by identifying factors that reactivate the silenced gene and restore production of the missing FMRP protein.
Healx Drug Repurposing Programme for Fragile X Syndrome
David Brown, MD, PhD, Ivan Angulo-Herrera, PhD and Anthony Hall of Healx present about the Drug Repurposing Programme for Fragile X syndrome.
MicroRNA Mediated Astroglial GLT1 Dysregulation in Fragile X
The team studied how glial cells, especially astrocytes, affect Fragile X. They tested microRNAs to restore GLT1 and reduce excess glutamate linked to brain hyperexcitability.
Aripiprazole (Abilify) in the Treatment of People with Fragile X: An Anecdotal Account
This article discusses Abilify (aripiprazole) as a “off-label” treatment for people with Fragile X syndrome (FXS). Abilify targets irritability, aggression, self-injury and severe tantrums.
Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases
In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Program and Fabio C. Tucci, PhD, CEO and co-founder at Epigen Biosciences.
Scientists Find a New Way to Reverse Symptoms of Fragile X
MIT Professor Mark Bear and colleagues have identified a new target for Fragile X therapeutics: GSK3 alpha. Several FRAXA research teams previously identified GSK3 beta as a treatment target.
Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast
In this episode Dr. Patrick Short talks to Dr. Bruce Bloom, CCO of Healx, and Dr. Mike Tranfaglia, CSO of FRAXA. about drug repurposing, machine learning, and Fragile X syndrome.
Considering Available Drugs for Fragile X: My Favorite Combination (So Far)
Which of the available drugs are best for managing fragile X syndrome? Most drugs have “off-target” effects which can have key advantages in some cases.
Less Active Immune System Evident in Fragile X Patients, Study Suggests
People with Fragile X syndrome are more likely to develop infections but less susceptible to autoimmune disorders than the overall population, a study found.
Results Reported: Using EEG Responses to Sound for Fragile X Drug Discovery
An obstacle in FXS research has been lack of reliable, unbiased biomarkers to assess herapies. This team found striking similarity in EEG biomarkers between mice and humans.
FRAXA Biotech Games, It Can Only Happen in an Open Community
The FRAXA Biotech Games lit up Cambridge Crossing with biotech teams uniting to raise funds and awareness for Fragile X research.
NH Fragile X Awareness Day Proclaimed at July Jam Fundraiser
The Fragile X July Jam in Derry, NH raised $6,000 for FRAXA! Gov. Sununu’s office also issued a proclamation for Fragile X awareness.
20 Landmarks Will Light Up for Fragile X Awareness Day
20 landmarks in the US & Canada will light up teal on July 22 for International Fragile X Awareness Day, spreading hope and awareness!
A Day in the Lab with FRAXA Investigator Dr. Tue Banke
Recently Laurie Bowler and her 19-year-old son Casey, who has Fragile X syndrome, visited FRAXA research grant recipient Dr. Tue Banke at his University of Washington laboratory. We hope you enjoy Laurie’s wonderful description of their adventure! FRAXA awarded $90,000 to Dr. Banke to study the Developmental Profile of Glutamatergic Synapses in Fragile X.
FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says
Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms that anxiety – not lack of interest – often hold them back.
Screening 2,320 FDA-Approved Drugs for Potential Treatment of Fragile X
FRAXA funded a screen of 2,320 FDA-approved compounds in the Fragile X fly model to identify hits that improve memory and social behavior for advanced testing.