Research aimed at finding Fragile X syndrome treatments is exploding. Why are so many pharmaceutical and biotech companies investing in this orphan indication? FRAXA chief scientific officer Dr. Michael Tranfaglia explains the many reasons Fragile X is such a hot topic.
Read moreResearch Updates
How FRAXA Prioritizes Research, Explained
![How FRAXA Prioritizes Research, Explained](https://www.fraxa.org/wp-content/uploads/2022/04/Research-Pyramid-Explained-720x300.png)
Dr. Mike Tranfaglia explains how FRAXA prioritizes research and the importance of looking at research from multiple angles. “It’s not either-or. It’s not we have a definitive treatment or we have a new drug treatment or we have a repurposing treatment. We can have all of those things, mixed or matched, in a personalized medicine kind of way and I think that’s what we’re headed for.”
Read moreAggression, Other Fragile X Behaviors Tend to Ease Over Teenage Years
![Aggression, Other Fragile X Behaviors Tend to Ease Over Teenage Years](https://www.fraxa.org/wp-content/uploads/2022/04/Aggression-Other-Fragile-X-Behaviors-Tend-to-Ease-Over-Teenage-Years-700x300.png)
Behavioral problems such as hyperactivity and aggression are generally more frequent in younger children with Fragile X syndrome, becoming less common as they grow through adolescence and journey toward adulthood, researchers report.
Read moreWhat FRAXA Is Excited about in the Upcoming Fragile X Research Grants, Explained
![What FRAXA Is Excited about in the Upcoming Fragile X Research Grants, Explained](https://www.fraxa.org/wp-content/uploads/2022/03/FRAXA-Explained-Excited-About-720x300.png)
Dr. Mike Tranfaglia shares what FRAXA is excited about as we work through reviewing all of the submitted Fragile X research grant applications. We find it especially exciting that so many new clinical trials are starting right now, as our major emphasis is getting the drugs and other treatment strategies that we have tested in the Fragile X mouse model to patients in clinical trials.
Read moreGordon Research Conference for Fragile X and Autism-Related Disorders, Explained
![Gordon Research Conference for Fragile X and Autism-Related Disorders, Explained](https://www.fraxa.org/wp-content/uploads/2022/03/Explained-GRC-yt-720x300.png)
The Fragile X and Autism-Related Disorders Gordon Research Conference is a biannual event that provides an international forum for the presentation and discussion of frontier research in these conditions. Dr. Mike Tranfaglia explains why this is the premier conference for researchers and the scientific community.
Read moreRecruiting: Clinical Study of Non-Invasive EEG for Children Ages 2-7
![Recruiting: Clinical Study of Non-Invasive EEG for Children Ages 2-7](https://www.fraxa.org/wp-content/uploads/2018/03/eeg1-576x300.png)
Dr. Carol Wilkinson, MD PhD, and Dr. Charles Nelson, PhD, at Boston Children’s Hospital are recruiting children ages 2-7 years with Fragile X syndrome to participate in a study of brain differences using non-invasive EEG.
Read moreCorrecting Fragile X Syndrome Deficits by Targeting Neonatal PKCε Signaling in the Brain
![Correcting Fragile X Syndrome Deficits by Targeting Neonatal PKCε Signaling in the Brain Banerjee team at College of Staten Island](https://www.fraxa.org/wp-content/uploads/2017/04/Banerjee-team-e1644863846647-720x300.jpg)
With this $90,000 grant from 2017-2018, Dr. Banerjee’s team has shown that enhancing PKCε can correct brain development and abnormal behaviors in Fragile X knockout mice and had their findings published in PubMed.
Read morePotential Upcoming Advances in Fragile X Research
![Potential Upcoming Advances in Fragile X Research](https://www.fraxa.org/wp-content/uploads/2022/02/Sonenberg-Kind-fi-720x300.png)
Dr. Peter Kind, Director of the Patrick Wild Centre and Professor of Developmental Neuroscience at the University of Edinburgh, and Dr. Nahum Sonenberg, James McGill professor of biochemistry at McGill University, share their optimism about the next 10 years of Fragile X research. They discuss where they think the next big discoveries will emerge.
Read moreNeurodevelopmental Drug Development Summit Presentation
![Neurodevelopmental Drug Development Summit Presentation](https://www.fraxa.org/wp-content/uploads/2022/01/NDD-Summit-fi-720x300.png)
FRAXA president and co-founder, Katie Clapp was one of three patient advocacy leaders invited to kick off the Neurodevelopmental Drug Development Summit with a presentation on Fragile X, and FRAXA Scientific Advisor, Dr. Elizabeth Berry-Kravis also presented lessons learned from clinical trials in Fragile X Syndrome.
Read moreReactivating the Fragile X Gene in Young Mice Reverses Symptoms
![Reactivating the Fragile X Gene in Young Mice Reverses Symptoms](https://www.fraxa.org/wp-content/uploads/2021/12/CRISPR-e1640663720464-720x300.jpg)
A new FRAXA-funded research project offers hope that Fragile X syndrome could be treated by reactivating the gene which is shut down in people with the syndrome. Researchers at the University of California, Riverside report that they were able to reduce FXS symptoms by inserting the FMR1 gene into the brains of very young mice.
Read more10 Year Vision for Collaborations That Transform Fragile X and Autism Research
![10 Year Vision for Collaborations That Transform Fragile X and Autism Research](https://www.fraxa.org/wp-content/uploads/2021/09/YouTube-Thumbnail-720x300.png)
The future offers hope for people living with Fragile X syndrome. Collaborations between the Fragile X community and other disability organizations help to provide understanding and advancement of research to bring effective treatments to families. FRAXA’s Dr. Mike Tranfaglia talks with Autism Science Foundation’s Allison Singer about the importance of their collaboration as we look forward to the next 10 years.
Read moreNew Fragile X Clinical Trial Announced by Healx
![New Fragile X Clinical Trial Announced by Healx](https://www.fraxa.org/wp-content/uploads/2021/10/artificial-intelligence-e1634834011962-720x300.png)
Healx’s AI-driven approach makes finding the right combination therapies more efficient, cost-effective, and rapidly ready for testing at FRAXA-DVI. It was this process that has brought Healx to its recent announcement sharing that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).
Read moreCorrecting the Brain’s Emotional Memory Center
![Correcting the Brain’s Emotional Memory Center](https://www.fraxa.org/wp-content/uploads/2021/10/Correction-of-Amygdalar-Dysfunction-in-Rat-Model-720x300.jpg)
Cell Reports has published the results of the study “Correction of Amygdalar Dysfunction in Rat Model of Fragile X Syndrome” by FRAXA Investigator Dr. Sumatra Chattarji and his team at the National Centre for Biological Sciences in Bangalore, India. The researchers investigated the synaptic basis of deficient conditioned fear and its reversal in Fragile X syndrome rats.
Read moreMaking Drug Development Efficient Through Community-Based Collaboration
![Making Drug Development Efficient Through Community-Based Collaboration](https://www.fraxa.org/wp-content/uploads/2021/10/Making-drug-development-efficient-webinar-720x300.png)
FRAXA co-founders Katie Clapp and Mike Tranfaglia, spoke virtually at the 5th Pharma Pricing Reimbursement and Market Access 2021 conference.
In this session, facilitated by Nadia Bodkin, PharmD, MS, from the Rare Advocacy Movement, Katie and Mike were joined by Christopher U. Missling, PhD, President and CEO of Anavex Life Sciences Corp. to discuss the collaboration between FRAXA and Anavex as a case study example to help raise awareness amongst others in the rare disease industry of these types of collaborations between advocacy and industry.
Read more2021 Fragile X Research Grants Funded by FRAXA Research Foundation
![2021 Fragile X Research Grants Funded by FRAXA Research Foundation](https://www.fraxa.org/wp-content/uploads/2021/09/Antonella-Borreca-Zaira-Boussadia-e1633102057303-720x300.jpeg)
Each year, FRAXA funds a diverse portfolio of research. Our FRAXA Fellowships are seed funding for the future, the feedstock for the Fragile X treatment development pathway. While we are looking to promote as many promising new approaches as possible, prominent themes emerge each year, as scientists around the world tackle previously neglected areas.
Read more20 Years of Advancing Fragile X Research: Progress Toward a Cure
![20 Years of Advancing Fragile X Research: Progress Toward a Cure](https://www.fraxa.org/wp-content/uploads/2021/09/WuXi-Mark-Bear-Advancing-FX-Research-fi-720x300.png)
Dr. Mark Bear joined the Fragile X field in 1999 when he received a research grant from FRAXA Research Foundation. At the time, we recognized the symptoms of Fragile X, and we knew its cause: a single missing protein. But we knew very little else. Dr. Bear traces the discoveries that now give us great optimism of finding effective treatments and ultimately a cure for Fragile X.
Read moreBrain Organoids, Moving Fragile X Research Forward
![Brain Organoids, Moving Fragile X Research Forward](https://www.fraxa.org/wp-content/uploads/2021/09/88100706_m-720x300.jpg)
There are many ways research produces discoveries, and all of them include a process of steps that build on each other. When an exciting new avenue appeared with potential for Fragile X syndrome, FRAXA stepped up to fund it. We now see the results of this grant and are excited to share them with you. The importance of different types of models have been shared and discussed over many years. We are now adding a “brain organoid” model to this group, and the potential behind it is really exciting.
Read moreTetra’s Fragile X Clinical Trial – The Most Successful So Far
![Tetra’s Fragile X Clinical Trial – The Most Successful So Far](https://www.fraxa.org/wp-content/uploads/2021/09/Mark-Gurney-FX-Trial-WuXi-webinar-fi-720x300.png)
Dr. Mark Gurney, CEO of Tetra Therapeutics, discusses how one of the earliest clues to the biology of Fragile X led to the most successful Fragile X clinical trial to date. FRAXA and Tetra began working together after a key FRAXA-funded study caught the attention of Dr. Gurney. Through the FRAXA Drug Validation Initiative, Dr. Patricia Cogram was able to conduct preclinical validation experiments with Tetra’s lead compound in record time, paving the way for clinical trials.
Read morePromising Results of Preclinical Study of ANAVEX®2-73
![Promising Results of Preclinical Study of ANAVEX®2-73](https://www.fraxa.org/wp-content/uploads/2021/08/anavex-header-507x300.png)
We are excited to share that Anavex Life Sciences announced today that preclinical data of the ANAVEX®2-73 (blarcamesine) study in Fragile X syndrome were published in the peer-reviewed journal, Scientific Reports.
Read moreSynaptogenix Announced Intention to Launch a Fragile X Clinical Trial with Bryostatin
![Synaptogenix Announced Intention to Launch a Fragile X Clinical Trial with Bryostatin Fragile X Clinical Trials](https://www.fraxa.org/wp-content/uploads/2021/08/Fragile-X-Clinical-Trials-e1628272789327-720x300.jpg)
One of the most exciting kinds of work that FRAXA does is following the journey of an experimental new treatment until it is ready for trials in people with Fragile X. From an initial idea, through the development process, to clinical trials, FRAXA helps out all along the way. From an initial idea, through the development process, to clinical trials, FRAXA helps out all along the way. The recent announcement by Synaptogenix is a great example of how FRAXA funding and use of FRAXA-DVI can accelerate research on Fragile X.
Read moreFragile X Syndrome: In Pursuit of a Cure Webinar
![Fragile X Syndrome: In Pursuit of a Cure Webinar](https://www.fraxa.org/wp-content/uploads/2021/06/In-Pursuit-of-a-Cure-for-Fragile-X-Syndrome-WuXi-AppTec-webinar-720x300.png)
A global webinar titled “Fragile X Syndrome: In Pursuit of a Cure,” took place on July 22, 2021 to commemorate World Fragile X Day. This complimentary event is co-organized with WuXi AppTec. We are delighted that more than 5,000 registered from more than 50 countries worldwide, coming together to raise awareness of Fragile X, and to foster collaborations towards effective treatments and ultimately a cure.
Read moreDrug Tolerance in MGluR5 Clinical Trials – Dr Patrick McCamphill 1:1 with FRAXA
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We have long suspected that the clinical trials of mGluR5 blockers from Novartis and Roche failed because the drug triggered tolerance, losing effect over time. With a $90,000 grant from FRAXA, Dr. Patrick McCamphill, a Postdoctoral Fellow in the MIT lab of Dr. Mark Bear, is investigating. He does indeed find tolerance, and now he is looking for ways to overcome it.
Read morePivotal Phase 3 Trial of Zygel in Severe Fragile X Possible This Year
![Pivotal Phase 3 Trial of Zygel in Severe Fragile X Possible This Year](https://www.fraxa.org/wp-content/uploads/2021/05/zynerba-bg-720x300.jpg)
Zynerba Pharmaceuticals reported receiving advice from the U.S. Food and Drug Administration (FDA) on the design of an upcoming Phase 3 clinical trial meant to confirm previous trial findings supporting Zygel as a cannabidiol treatment in a specific subset of Fragile X syndrome patients. The new trial, called RECONNECT, is expected to launch before October, and will mainly enroll children and adolescents with a complete (100%) methylation of FMR1, the gene mutated in Fragile X.
Read moreTetra Releases Full Results of FRAXA-Funded Clinical Trial of PDE4D Inhibitor
![Tetra Releases Full Results of FRAXA-Funded Clinical Trial of PDE4D Inhibitor](https://www.fraxa.org/wp-content/uploads/2020/03/AimeAndJaxson-e1584753971822-720x300.jpg)
Today, Tetra Therapeutics published the full results of its PDE4D trial published the full results to their announcement. Now having reviewed the full results, FRAXA can confidently say that the PDE4D drug trial gives hope to patients and families that Fragile X Syndrome is a treatable disorder, and this particular drug can improve intellectual disability.
Read moreParkinson’s Therapy May Hold Promise for Fragile X
![Parkinson’s Therapy May Hold Promise for Fragile X](https://www.fraxa.org/wp-content/uploads/2021/02/FXNews-e1614182670721-720x300.jpg)
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.
“One of the most intriguing things about this study is that it points to an entire drug class (not just the one drug used) as potentially therapeutic for Fragile X. Many available compounds block A2A receptors, and we know they are safe and effective.
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