New Partners Aim to Accelerate the Discovery and Repurposing of Medicines for Rare Neurological Diseases
Healx has launch a global Rare Treatment Accelerator program to tackle Fragile X syndrome and 39 other rare diseases. It all started with a small FRAXA grant to repurpose available drugs for Fragile X.
Ganaxolone Fragile X Clinical Trial Showed Disappointing Results
Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.
FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says
Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms that anxiety – not lack of interest – often hold them back.
Fragile X Research has More Runners in the Race
We asked for your help to find effective treatments and a cure for Fragile X, and you came through for us. Thank you all so much! FRAXA’s 2018 year-end annual appeal raised $733,017 to fund research!
Tetra Announces $40M to Advance BPN14770 for FXS and Alzheimer’s Disease
Tetra conducted a Phase 2 study of BPN14770 in adults with Fragile X Syndrome. This clinical trial was made possible by early work with the FRAXA-DVI and over $200,000 from FRAXA.
Drug Duo Delivers Brain, Behavioral Benefits for Fragile X Syndrome
Administering a cholesterol drug alongside an antibiotic eases atypical behavior and restores the signaling balance in the brains of people with fragile X syndrome.
Spectrum News – Newly Discovered Aspects of Fragile X Spur Next Wave of Drugs
Many drugs for Fragile X syndrome have failed in large clinical trials, but candidates that target new aspects of the condition may fare better.
Non-Invasive Imaging as a Biomarker for Fragile X Clinical Trials
FRAXA funded MRI research to track brain connectivity changes in Fragile X. This could yield objective biomarkers for testing treatments in mice and humans.
Mega Green Tea Extract to Treat Fragile X?
FRAXA funded a clinical trial of EGCG (green tea extract) on cognitive function in adults with FXS. The team in Barcelona reported memory, attention, and mental flexibility improvements.
How Promising is CRISPR for Fragile X?
FRAXA Investigator Peter Todd, MD, PhD, tells us about CRISPR in Fragile X research, how realistic is it that it could turn the Fragile X gene back on, and if it can really cure Fragile X.
Retinoic Acid Signaling is Blocked by Fragile X Mutation
This FRAXA-funded team at Stanford University found that the Fragile X mutation impairs homeostatic plasticity in human neurons, by blocking synaptic retinoic acid signaling.
Aripiprazole as a Treatment for Fragile X Syndrome
Years ago FRAXA funded Dr. Craig Erickson to run a trial of aripiprazole (aka Abilify). FRAXA guest writer Hannah Miles recently caught up with Dr. Erickson to learn the results of the trial.
Study Examines Behavioral Traits of Fragile X Patients Without Autism
Patients with Fragile X syndrome who don’t meet the cut-off for a diagnosis of autism show a decrease in impulsivity and repetitive questioning over time, when compared with patients who do, a new study shows.
Lysogene Partners with FRAXA Investigator Dr. Hervé Moine to Develop Gene Therapy for Fragile X
Lysogene, a French biopharmaceutical company working to develop gene therapy treatments for brain disorders, is partnering with FRAXA Investigator Dr. Herve Moine to tackle Fragile X.
FRAXA Research Grants Drive Big Investments in Fragile X
FRAXA is working with hundreds of university labs and more than 30 pharmaceutical companies around the world. Dr. Michael Tranfaglia spends a lot of his time advising and collaborating with industry partners.
Can CRISPR Cure Fragile X Syndrome?
CRISPR/Cas9 was used by MIT researchers to remove the molecular tags that keep the mutant gene shut off in Fragile X syndrome neurons and resulted in some of them producing protein normally.
CRISPR Reactivation of the Fragile X Gene
Using CRISPR to reactivate FMRP, “We are trying to target the first event that goes wrong in Fragile X syndrome”, says FRAXA Investigator, Dr. Peter Todd.
Newly Discovered Regulatory Pathways in Fragile X
Studies at Yale University and elsewhere show FMRP plays a significant role in regulation of potassium channels. Potassium channel opener drugs could rescue some symptoms of Fragile X.
Drug Repurposing Study Results Accelerate Progress Towards Fragile X Treatments
Drug repurposing leverages the detailed information available on approved drugs and reduces the time and money needed to deliver safe “new” treatments, with greater success rates and quicker impact.
In Their Own Words: Reports From the International Fragile X Workshop
The 18th International Fragile X Workshop in Quebec was a great success, featuring more Fragile X research than ever before!
Brain Imbalance Target of Dr. Erickson’s New Clinical Trial
Dr. Erickson’s trial targeted brain imbalance in Fragile X by boosting GABA signaling with AZD7325, aiming to restore excitatory–inhibitory balance and improve symptoms.
Fragile X in the Forefront of International Conference
The 18th International Fragile X Workshop kicks off in Quebec, uniting global scientists to share breakthroughs in Fragile X and ASD.
Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials
Once a neural marker is identified for a particular challenge, it can be measured during drug and behavioral therapy trials to see if a child is improving based on objective biological measures.
Targeting Serotonin Receptors to Treat Behavioral and Psychological Symptoms
Dr. Clinton Canal targets serotonin receptors to correct brain signaling in Fragile X, guiding drug discovery and repurposing of therapies for anxiety, seizures, and behavior.