Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice
Treating Fragile X might require a combination of drugs. FRAXA-DVI tested ibudilast and gaboxadol in Fragile X mice. Together they rescued a wide array of symptoms.
Coming Together for Rare Disease Day 2023
Today we mark Rare Disease Day. FRAXA is committed to advancing research on Fragile X, one of the most common rare diseases worldwide.
World Fragile X Day Is Lighting the Way for Fragile X Research
On July 22, 383 landmarks worldwide lit up for World Fragile X Day—shining bright to raise awareness and celebrate research progress.
New Fragile X Clinical Trial Announced by Healx
Healx has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for a Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).
Fragile X Syndrome: In Pursuit of a Cure Webinar
Global webinar “Fragile X Syndrome: In Pursuit of a Cure” on July 22, 2021 commemorated World Fragile X Day. Over 5,000 registered from more than 50 countries.
2021 FRAXA Awards – Recognizing Perseverance and Dedication
In conjunction with World Fragile X Day 2021, FRAXA Research Foundation recognizes its annual award recipients. We are fortunate to partner with these individuals for Fragile X families.
Pivotal Phase 3 Trial of Zygel in Severe Fragile X Possible This Year
Zynerba Pharmaceuticals received advice from the U.S. Food and Drug Administration (FDA) on the design of a Phase 3 clinical trial of Zygel as a cannabidiol treatment for Fragile X syndrome.
Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model
Long-term, but not short-term, treatment with bryostatin-1 arrested some behavioral and cognitive symptoms in a mouse model of Fragile X.
Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest
Zynerba presented Fragile X clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel.
Healx Drug Repurposing Programme for Fragile X Syndrome
David Brown, MD, PhD, Ivan Angulo-Herrera, PhD and Anthony Hall of Healx present about the Drug Repurposing Programme for Fragile X syndrome.
Mechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse
We hear from Devin K. Binder, MD, PhD, Professor, University of California at Riverside Medical School and Khaleel Razak, PhD, Professor, University of California at Riverside.
Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases
In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Program and Fabio C. Tucci, PhD, CEO and co-founder at Epigen Biosciences.
Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast
In this episode Dr. Patrick Short talks to Dr. Bruce Bloom, CCO of Healx, and Dr. Mike Tranfaglia, CSO of FRAXA. about drug repurposing, machine learning, and Fragile X syndrome.
Less Active Immune System Evident in Fragile X Patients, Study Suggests
People with Fragile X syndrome are more likely to develop infections but less susceptible to autoimmune disorders than the overall population, a study found.
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.
Healx Raises $56M to use AI to Find Treatments for Fragile X & Other Rare Diseases
Healx has secured $56M in new financing to build a clinical-stage portfolio for rare diseases, including treatments for Fragile X syndrome, and to launch a global Rare Treatment Accelerator program. Where the traditional drug discovery model takes more than a decade and can run into the billions of dollars, Healx’s AI-driven approach makes the process faster, more efficient and cost-effective.
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.
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.
Repurposing Study II: Evaluating Combinations of Drugs to Treat Fragile X
FRAXA partnered with Healx to use AI to find approved drugs and drug combos that could treat Fragile X. Top candidates are now being tested in Fragile X models.
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.
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.
University of Cambridge Startup Healx is Rapidly Identifying Existing Drugs to Help Fragile X Patients
FRAXA awarded $44,000 to Healx for drug repurposing to find new treatments for Fragile X syndrome. The results include eight top "hits" which show promise for Fragile X.