Integrating Human and Mouse Studies in Fragile X Syndrome – an NIH Center Approach
Presentations by Craig Erickson, Ernest Pedapati, Devin Binder, and Kimberly Huber about their research on Fragile X as part of their NIH Center of Excellence.
Developing Arbaclofen for Fragile X – Dr. Mark Bear 1:1 with FRAXA
MIT professor Dr. Mark Bear founded Allos Pharma to develop arbaclofen (STX209) as a treatment for Fragile X syndrome. Dr. Bear sat down with FRAXA co-founder Katie Clapp to share the story and next steps.
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.
Interrogate the Functions of FMRP in Brain Development Using Stem Cells
Dr. Xinyu Zhao of the Waisman Center and Department of Neuroscience at University of Wisconsin-Madison joins us in this seminar to present Interrogate the Functions of FMRP in Brain Development Using Stem Cells.
Sincere Gratitude for a Successful #GivingTuesday 2020
From the bottom of our hearts—thank you! With 150+ donors, FRAXA raised $70,047 on #GivingTuesday. Your support brings hope to Fragile X families.
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.
FRAXA Biotech Games™ 2020 Won by Ginkgo Bioworks
The FRAXA Biotech Games united the biotech community for fun, networking, and collaboration—raising funds for a great cause!
Yale Researcher Dr. Elizabeth Jonas 1:1 with FRAXA
We talk with Dr. Elizabeth Jonas at Yale School of Medicine about her new research suggesting that leaky mitochondria which may be involved in Parkinson’s and Fragile X syndrome.
MicroRNAs as Biomarkers in Fragile X Syndrome
The team at Johns Hopkins University studied groups of small RNAs, known as microRNAs, which are greatly decreased in brain tissue of Fragile X mice vs. normal controls.
Auditory System Dysfunction and Drug Tolerance in the Fragile X Mouse
A $90K FRAXA grant will help uncover why Fragile X causes sound hypersensitivity and test ways to correct brain circuit dysfunction linked to auditory overload.
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.
Mechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse
We hear from Devin K. Binder, MD, PhD, Professor, University of California at Riverside Medical School and Khaleel Razak, PhD, Professor, University of California at Riverside.
A Tribute to Doris Buffett, Philanthropist
Doris Buffett, sister of Warren Buffett, passed at 92. She donated over $100M and called FRAXA “the gold standard” in grassroots charities.
Fragile X Community Launches #TealTakeover
From a few Zoom calls came an idea to celebrate Fragile X Awareness Day with three online events: the #TealTakeover!
Talking Step Up for Fragile X
Thanks to Talk Fragile X for featuring FRAXA’s Ellen Skala! She shared exciting details about our virtual event, Step Up for Fragile X!
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.
Landmarks Light Up Fragile X Awareness Day 2020
On Fragile X Awareness Day 2020, 67 landmarks across the US & Canada lit up teal! Explore the map & celebrate this growing global movement.
Talk Fragile X Podcast with Dave Bjork
Thank you, Talk Fragile X, for featuring FRAXA’s Dave Bjork on your podcast! We’re grateful for the chance to share our mission and research progress.
Trofinetide Clinical Trial Results Published
Neuren Pharmaceuticals has published results of a Phase 2 Fragile X Clinical Trial of Trofinetide ih Fragile X syndrome. This trial tested a new design which can be used in future trials.
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.