Fragile X syndrome research shows PDE inhibitors may improve brain function and behavior, with promise for related neurodevelopmental disorders.
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Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
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 moreAdvancing 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.
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