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

November 23, 2020

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.

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

February 4, 2019

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.

Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases

July 15, 2020

This is the first in a series of webinars focused on current topics in Fragile X research. In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Programand Fabio C. Tucci, PhD, Chief Operating Officer and co-founder at Epigen Biosciences, Inc.

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

October 22, 2020

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.

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

February 10, 2020

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.

Study Examines Behavioral Traits of Fragile X Patients Without Autism

June 21, 2018

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.

Forbes, “Clinical Trials Expand For AI-Designed Drug To Treat Fragile X Syndrome”

October 20, 2021

British startup Healx has secured FDA approval for a phase 2a clinical trial of an AI-discovered compound that could help manage the symptoms of the genetic disorder Fragile X syndrome. The start of the trial marks another milestone in the use of artificial intelligence to help find new applications for existing drugs by mining patient records and research databases.

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

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

Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases

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

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

Study Examines Behavioral Traits of Fragile X Patients Without Autism

Forbes, “Clinical Trials Expand For AI-Designed Drug To Treat Fragile X Syndrome”

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