Fragile X News Today, Author at Fragile X Research

Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model

Fragile X News Today FRAXA Updates November 23, 2020Alzheimer's, autism, FDA, GABA, mGluR5

Long-term, but not short-term, treatment with bryostatin-1 — Neurotrope’s lead investigational therapy — arrested such behavioral and cognitive symptoms as hyperactivity, difficulties with daily life activities, and learning and memory deficits in a mouse model of Fragile X syndrome.

Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest

Fragile X News Today Research Updates October 22, 2020October 22, 2020CBD, FDA

Zynerba presented clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel, may reduce behavioral abnormalities in children with Fragile X syndrome who have more severe disease.

Companies Move to Advance Potential Cognitive Treatment for Fragile X

Fragile X News Today FRAXA Updates March 13, 2020Alzheimer's, BPN14770, FDA, NIH, PDE4, Tetra Therapeutics

Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits, including Fragile X syndrome and Alzheimer’s disease. The agreement builds on an earlier collaboration between the two companies, and aims to further accelerate BPN14770’s development and potential marketing. It is currently in clinical testing in both Fragile X and Alzheimer’s patients.

Less Active Immune System Evident in Fragile X Patients, Study Suggests

Fragile X News Today Research Updates February 10, 2020February 10, 2020autism, seizure

People with Fragile X syndrome are more likely to develop infections, but are less susceptible to autoimmune disorders than the overall population, a new study found. Taken together, this suggests that the immune system is underactive in this patient population. The study, titled, “The phenotypical implications of immune dysregulation in Fragile X syndrome,” was published in the European Journal of Neurology.

New Partners Aim to Accelerate the Discovery and Repurposing of Medicines for Rare Neurological Diseases

Fragile X News Today FRAXA Updates December 13, 2019Healx

First Healx secured $56M in new financing to launch a global Rare Treatment Accelerator program to tackle Fragile X syndrome and 39 other rare diseases. Now they have built a partnership with Boehringer Ingelheim worth millions. It all started with a small FRAXA grant to Healx to repurpose available drugs for Fragile X.

FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says

Fragile X News Today Cincinnati Children's Hospital • Erickson, Craig • Research Updates February 4, 2019AstraZeneca, autism, AZD7325

Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms what so many parents, caretakers, and educators suspect: people with fragile X want to be social, and it is anxiety – not lack of interest – which usually hold them back. If anxiety could be reduced, more sociability would likely follow. Dr. Erickson is a Fragile X expert and FRAXA investigator who is currently conducting a Fragile X clinical trial of an investigational new drug.

Study Examines Behavioral Traits of Fragile X Patients Without Autism

Fragile X News Today Research Updates June 21, 2018February 4, 2019autism, NIH

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.