Opening the Gate: BK Channel Modulation as a Promising Treatment Strategy for Fragile X Syndrome
Fragile X syndrome research is opening the door to BK channel therapies that may calm hyperexcitable brain circuits and improve key symptoms.
Cognitive Rigidity: a Hallmark of Fragile X and Autism
Cognitive rigidity shapes daily life in Fragile X, driving anxiety and repetitive behaviors. Learn its impact on treatment and independence.
Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration
FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.
BK Channel Openers: A New Drug for Fragile X Is Ready for Clinical Trials
A promising new BK channel opener, SPG601 from Spinogenix, is entering clinical trials for Fragile X syndrome. Learn about its potential to restore synaptic function and address core symptoms.
Pioneering 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.
Innovative 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.
Unraveling 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.
Breakthrough 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.
Fragile 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.
Considering Available Drugs for Fragile X: My Favorite Combination (So Far)
Which of the available drugs are best for managing fragile X syndrome? Most drugs have “off-target” effects which can have key advantages in some cases.
Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives
FRAXA is identifying existing, approved drugs that could be repurposed for Fragile X, allowing potential treatments to move faster and at lower risk than starting from scratch.
CRISPR – Does it hold promise for Treatment of Fragile X Syndrome?
CRISPR offers the tantalizing possibility of “editing” genes precisely, and it could (theoretically) excise the methylated trinucleotide repeat sequence from Fragile X cells, returning them to normal.
FRAXADev – Developing BK Channel Openers for Fragile X Syndrome
FRAXADev is an initiative in France not connected with FRAXA Research Foundation. The name is coincidental. FRAXADev seeks to develop a new kind of drug for Fragile X.
Fragile X: Past, Present, Future – Video
Michael Tranfaglia, MD, FRAXA Medical Director discussed how research has brought us to the point of clinical trials, the problems encountered in recent trials, and where we go from here.
Fragile X Treatment: New Research Directions
In the wake of negative results from several high-profile clinical trials in Fragile X, we find ourselves questioning many of our previous assumptions about the nature of this disorder.
Neuren’s NNZ-2566 Shows Clinical Benefit in Rett Syndrome Trial
This isn’t a Fragile X trial, but the Neuren compound, NNZ-2566, that is in trials now for Fragile X has shown significant positive effects in a Phase 2 trial for Rett syndrome.
Roche reports clinical trial negative results
Roche has shared the sad news that their clinical trials in Fragile X have been unsuccessful. They hosted a Webcast to explain the results.
Why Did Fragile X Clinical Trials of mGluR Antagonists Fail?
by Michael Tranfaglia, MD. In my opinion, the Fragile X clinical trials of AFQ056 sponsored by Novartis failed because of a dose range that was inadequate for Fragile X, and because of the unexpected development of tolerance.
Fragile X Syndrome Treatment Target: MMP-9
This paper shows that MMP-9 dysregulation is a critical part of the pathology of Fragile X, and MMP-9 should be considered a major treatment target for Fragile X syndrome.
Fragile X Clinical Trial: Novartis Trial Results Are In, and They’re Not Pretty
This year’s Gordon Conference just finished, and Novartis presented their results. To say that the trial results for AFQ056 were disappointing would be the understatement of the century!
What Treatments Work for FXTAS?
FXTAS affects many in our Fragile X community. Research aims to uncover its cause and guide more effective treatments.
FRAXA Progress and Future Plans
Huge momentum in Fragile X research: major results released, widespread media attention, and Phase III trials moving toward possible treatments.
What Works, and What Doesn’t
Early on, no one knew which path would work. Now the results are clear, and they’re directing FRAXA toward the next major Fragile X treatment breakthrough.
Compound that Inhibits mGluR5 Corrects Signs of Fragile X in Adult Mice
A Roche and MIT study published in Neuron finds that an mGlu5 inhibitor, CTEP, can reverse many Fragile X symptoms in adult mice.