Tetra Releases Full Results of FRAXA-Funded Clinical Trial of PDE4D Inhibitor
Today, Tetra Therapeutics published the full results of its PDE4D trial. The data from this phase 2 clinical trial suggests the PDE4D drug can improve intellectual disability in Fragile X syndrome.
Parkinson’s Therapy May Hold Promise for Fragile X
A study funded by FRAXA in Italy has encouraging results for people with Fragile X: drugs that block adenosine receptors (A2A) reversed signs of Fragile X in a mouse model.
The Why and How of FRAXA’s Work to Find a Cure for Fragile X
Thank you Talk Fragile X for having FRAXA’s cofounder Katie Clapp on your podcast! She shared our motivation for finding effective treatments and ultimately a cure for Fragile X.
Beneath the Surface of Fragile X Syndrome: Study Sheds Light on What’s Happening in Nerve Cells
Lynne Maquat discovered that many irregularities in cells that lack FMRP are due to misregulated nonsense-mediated mRNA decay, or NMD. Results published.
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.
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.
Overcoming the Placebo Effect in Fragile X Clinical Trials
In a placebo-controlled clinical trial, some participants are given an experimental medication, while others are given a placebo. A strong placebo effect can obscure results.
Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest
Zynerba presented Fragile X clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel.
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.
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.
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.
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.
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.
Companies Move to Advance Potential Cognitive Treatment for Fragile X
Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits.
Ketogenic Diet Eases Symptoms in Fragile X Male Mice
Westmark Lab is studying sleep patterns in Fragile X mice, working with UW-Madison to develop tools that speed EEG data analysis
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.
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.
Should You Participate in a Fragile X Clinical Trial?
A Fragile X clinical trial of a new PDE4D allosteric inhibitor from Tetra Therapeutics is nearly complete. The drug has a unique mechanism of action that might improve cognitive and memory function.