Explore how QurAlis and UMass Chan are revolutionizing Fragile X syndrome treatment using advanced ASO technology, setting new standards in therapeutic development.
Read moreResearch Updates
Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration
![Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration](https://www.fraxa.org/wp-content/uploads/2024/06/serotonin-and-silhouette-of-a-man-head-720x300.jpg)
A new treatment target for Fragile X syndrome has emerged from multiple research labs and a pharmaceutical startup. Neurolixis, a FRAXA pharma partner, has announced a new Fragile X development program.
Read moreExploring Advances Fragile X Research: Comprehensive Webinar Highlights – May 2024
![Exploring Advances Fragile X Research: Comprehensive Webinar Highlights – May 2024](https://www.fraxa.org/wp-content/uploads/2024/04/2024-05-Research-QA-part2-btn-720x300.png)
Discover the latest in Fragile X research with insights from our May 2024 webinar. Learn about new FRAXA grants, key clinical trials, and scientific updates that are shaping the future of Fragile X syndrome treatments.
Read moreBK Channel Openers: A New Drug for Fragile X Is Ready for Clinical Trials
![BK Channel Openers: A New Drug for Fragile X Is Ready for Clinical Trials](https://www.fraxa.org/wp-content/uploads/2024/05/Restoring-Synapses-BK-Channel-Opener-720x300.png)
Discover the promising new BK channel opener, SPG601, now entering clinical trials for Fragile X syndrome. Learn about its potential to restore synaptic function and address core symptoms.
Read moreRecruiting: Tetra Therapeutics Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
![Recruiting: Tetra Therapeutics Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome Tetra Therapeutics Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2024/02/Tetra-Therapeutics-Phase-3-Clinical-Trial-of-Zatolmilast-in-Fragile-X-Syndrome-720x300.png)
Tetra Therapeutics has launched large scale clinical trials of their phosphodiesterase (PDE) inhibitor for males ages 9-45 with Fragile X syndrome. FRAXA Research Foundation’s basic and translational research pointed the way to phosphodiesterase inhibitors to treat Fragile X.
Read moreRecruiting: Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option
![Recruiting: Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option](https://www.fraxa.org/wp-content/uploads/2022/11/RECONNECT-Virtual-Fragile-X-Clinical-Trial-Now-Enrolling-720x300.jpg)
Harmony Biosciences is now recruiting for the RECONNECT Phase 3 clinical trial of ZYN002, a potential treatment for Fragile X syndrome. The trial offers an at-home participation option, making it accessible for patients across the US, Australia, and the UK. Join us in this groundbreaking study and contribute to the future of Fragile X syndrome treatment.
Read moreRenewed Hope: Navigating Towards a Cure for Fragile X Syndrome
![Renewed Hope: Navigating Towards a Cure for Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2024/04/Sutton_Todd_Mills_Rodriguez_015-e1712066000308-720x300.jpg)
Discover how Dr. Peter Todd’s latest Fragile X Syndrome research offers hope for advanced treatments and a possible cure, marking a new era in FXS therapy.
Read moreInside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments
![Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments](https://www.fraxa.org/wp-content/uploads/2024/03/FRAXA-DVI-2024-6-720x300.jpg)
Explore how the FRAXA Drug Validation Initiative is revolutionizing Fragile X syndrome treatment, leading the charge towards innovative therapies and hope for affected families.
Read moreTwo-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice
![Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice](https://www.fraxa.org/wp-content/uploads/2023/02/Sex-specific-Learning-Differences-Found-in-Fragile-X-Patients-Mouse-Model-700x300.png)
Treating Fragile X syndrome will likely require a combination of drugs, as a single medication may not address all symptoms. At FRAXA-DVI, Dr. Patricia Cogram and her team recently tested a combination of two investigational new drugs in Fragile X mice, with support from Healx. Together ibudilast and gaboxadol rescued a wide array of symptoms in the mice.
Read moreAn Update from Harmony Biosciences on Giving Tuesday
![An Update from Harmony Biosciences on Giving Tuesday](https://www.fraxa.org/wp-content/uploads/2023/11/Jeffrey-M.-Dayno-720x300.jpg)
Harmony Biosciences recently completed its acquisition of Zynerba Pharmaceuticals. I am excited for us to progress their work in pursuit of a treatment option for people living with Fragile X syndrome.
Read moreFRAXA Research Foundation Partners with Autism BrainNet
![FRAXA Research Foundation Partners with Autism BrainNet](https://www.fraxa.org/wp-content/uploads/2023/08/FRAXA-Autism-BrainNet-720x300.png)
Discover how FRAXA Research Foundation’s collaboration with Autism BrainNet accelerates Fragile X syndrome research by collecting vital postmortem brain tissue. Dive into the significance of brain studies for deeper insights and potential therapeutic interventions.
Read morePioneering Community-Based Drug Development for Fragile X Syndrome
![Pioneering Community-Based Drug Development for Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/07/Steward-Lab-FRAXA-2-720x300.jpg)
Discover how FRAXA leverages Community-Based Drug Development to create impactful therapies for Fragile X syndrome. Join us as we reshape the future of rare disease treatment.
Read moreInnovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy
![Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy](https://www.fraxa.org/wp-content/uploads/2023/06/Richter-Shah-Berry-Kravis-Watts-Banbury-Meeting-720x300.jpg)
This changes everything! FRAXA funded research introduces Antisense Oligonucleotide (ASO) Therapy, redefining Fragile X syndrome treatment and understanding.
Read moreUnraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation
![Unraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation](https://www.fraxa.org/wp-content/uploads/2023/06/Hungoo-Lee-Jeannie-Lee-scaled-e1687181022594-720x300.jpg)
Discover groundbreaking methods for reactivating the FMR1 gene in Fragile X syndrome. Dive into the transformational research and the implications of self-healing at a cellular level.
Read moreAllos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen
![Allos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen](https://www.fraxa.org/wp-content/uploads/2020/11/397686_4212565235804_452910278_n-e1605762012898-720x300.jpg)
Discover Allos Pharma’s advancements in a pivotal Phase 3 trial for Fragile X syndrome treatment, Arbaclofen. Learn how their FDA-informed trial design might finally bring hope to the Fragile X community.
Read moreBreakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies
![Breakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies 2023 Fragile X Research Banbury Meeting (1)](https://www.fraxa.org/wp-content/uploads/2023/05/2023-Fragile-X-Research-Banbury-Meeting-1-720x300.jpg)
Explore the latest breakthroughs in Fragile X research unveiled at the recent Banbury Meeting. Discover novel strategies, from gene therapy to protein replacement, that bring hope for curative therapies.
Read moreFRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research
![FRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research](https://www.fraxa.org/wp-content/uploads/2023/05/Fragile-X-Biomarker-Research-hero-720x300.png)
Help accelerate research on Fragile X syndrome biomarkers by contributing samples to the COMBINEDBrain Consortium’s project. Contact Katie Clapp at FRAXA Research Foundation to learn how you can participate.
Read moreFragile X Syndrome and Cancer Research: Unexpected Links and Opportunities for Collaboration
![Fragile X Syndrome and Cancer Research: Unexpected Links and Opportunities for Collaboration](https://www.fraxa.org/wp-content/uploads/2023/04/Fragile-X-Syndrome-and-Cancer-scaled-e1680705942845-720x300.jpg)
Discover unexpected links between Fragile X Syndrome and cancer. Studies show people with Fragile X have much lower cancer rates. Explore new opportunities for collaboration in this promising research.
Read moreFRAXA Investigator Lynne Maquat Awarded 2023 Gruber Genetics Prize
![FRAXA Investigator Lynne Maquat Awarded 2023 Gruber Genetics Prize Lynne Maquat](https://www.fraxa.org/wp-content/uploads/2023/03/Lynne-Maquat-720x300.jpg)
Dr. Maquat, 2023 Gruber Genetics Prize winner, discovered NMD, a key surveillance system in the body that protects against mistakes in gene expression. Her groundbreaking research has led to new therapies for Fragile X syndrome.
Read moreSex-specific Learning Differences Found in Fragile X Patients, Mouse Model
![Sex-specific Learning Differences Found in Fragile X Patients, Mouse Model](https://www.fraxa.org/wp-content/uploads/2023/02/Sex-specific-Learning-Differences-Found-in-Fragile-X-Patients-Mouse-Model-700x300.png)
Girls and women with Fragile X syndrome show different learning impairments relative to boys and men with the disease, a finding that was paralleled in a mouse model of the disease, a study found.
Read moreA Look Back at 2022 and Ahead to 2023 Research Prospects
![A Look Back at 2022 and Ahead to 2023 Research Prospects](https://www.fraxa.org/wp-content/uploads/2023/01/Aubuchon-scaled-e1673544471591-720x300.jpeg)
This holiday season you proved that the adage “actions speak louder than words” is entirely accurate. We sincerely appreciate your gifts in action. FRAXA supporters came out in full force to meet the generous $100,000 challenge offered on Giving Tuesday.
Read more10 Year Vision for Fragile X Research – Dr. Elizabeth Berry-Kravis & Dr. Patricia Cogram
![10 Year Vision for Fragile X Research – Dr. Elizabeth Berry-Kravis & Dr. Patricia Cogram](https://www.fraxa.org/wp-content/uploads/2022/12/WuXi-Cogram-EBK-10-yr-fi-720x300.png)
In this video we hear from FRAXA Investigators Dr. Patricia Cogram, Professor at the University of Chile, and Dr. Elizabeth Berry-Kravis, Professor at Rush University Medical Center as they reflect on the progress that has been made and visualize what they see happening in the next 10 years for people living with Fragile X syndrome.
Read moreClinical Trial Results Reported for Phase 3 CONNECT-FX Study of Zygel™
![Clinical Trial Results Reported for Phase 3 CONNECT-FX Study of Zygel™](https://www.fraxa.org/wp-content/uploads/2021/09/zynerba-hero-scaled-e1631635334556-720x300.jpg)
Results have just been published from Zynerba Pharmaceuticals’s phase 3 clinical trial of Zygel™ in the Journal of Neurodevelopmental Disorders. In this trial, 212 children and adolescents aged 3 to 17 years were given Zygel or placebo for 12 weeks.
Read moreFRAXA Volunteer Participates in Peer Reviewed Medical Research Program for the Department of Defense
![FRAXA Volunteer Participates in Peer Reviewed Medical Research Program for the Department of Defense](https://www.fraxa.org/wp-content/uploads/2022/08/Frobish-Family-720x300.jpg)
FRAXA nominated advocate, Jennifer Frobish, recently evaluated research applications submitted to the Peer Reviewed Medical Research Program (PRMRP) of the Congressionally Directed Medical Research Programs (CDMRP).
Read moreWhy Pharma Companies Take on Fragile X, Explained
![Why Pharma Companies Take on Fragile X, Explained](https://www.fraxa.org/wp-content/uploads/2022/06/Why-Pharma-Companies-Take-on-Fragile-X-Explained-720x300.png)
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.
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