Shionogi’s EXPERIENCE clinical trials for Fragile X syndrome are nearing completion. Enrollment for the adult trial (EXPERIENCE-301) is now closed, while the adolescent trial (EXPERIENCE-204) is in its final phase. Learn more about the study, FRAXA’s role, and the open-label extension.
Read moreResearch Updates
Advancing Curative Therapy for Fragile X Syndrome: Turner Lab Secures $1M Grant
Fragile X syndrome research accelerates as the Turner Lab secures $1M to expand FRAXA-funded work on curative therapy targeting the missing FMRP protein.
Read moreFRAXA-Funded Research Explores ISRIB as a Potential Treatment for Fragile X
ISRIB for Fragile X syndrome is being studied as a potential treatment to restore brain function and social behavior. Researchers investigate its effects.
Read moreGEXVal and FRAXA Collaborate to Advance Fragile X Research with Phase 2a Trial
GEXVal and FRAXA collaborate to advance Fragile X research with the Phase 2a trial of GXV-001, supported by AMED’s funding program.
Read moreBK Channel Openers: A New Hope for Fragile X Treatment – Insights from Kaerus Bioscience CEO Robert Ring
Kaerus Bioscience’s BK channel openers for Fragile X syndrome are advancing through Phase 1 trials, offering hope for new treatments with FRAXA’s continued support.
Read moreNPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome
NPR spotlights zatolmilast, a promising drug offering new hope for individuals with Fragile X syndrome. Families report life-changing improvements in anxiety, communication, and independence.
Read moreMarvel Biosciences Partners with FRAXA to Test MB204 for Fragile X Syndrome
Discover how Marvel Biosciences and FRAXA Research Foundation are collaborating to test MB204, a promising new treatment for Fragile X syndrome, building on groundbreaking adenosine receptor research.
Read moreQurAlis and UMass Chan Advance Fragile X Syndrome Treatment using ASOs (Antisense Oligonucleotides)
Explore how QurAlis and UMass Chan are revolutionizing Fragile X syndrome treatment using advanced ASO technology, setting new standards in therapeutic development.
Read moreFragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration
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
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
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: Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option
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 moreFRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research
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 moreRenewed Hope: Navigating Towards a Cure for Fragile X Syndrome
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
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
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
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
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
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
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
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
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
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 moreFragile X Syndrome and Cancer Research: Unexpected Links and Opportunities for Collaboration
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
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.
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