Fragile X News Today, Author at Fragile X Research

Pivotal Phase 3 Trial of Zygel in Severe Fragile X Possible This Year

Fragile X News Today Research Updates May 17, 2021November 22, 2021CBD, endocannabinoid, FDA, NIH, Zygel

Zynerba Pharmaceuticals reported receiving advice from the U.S. Food and Drug Administration (FDA) on the design of an upcoming Phase 3 clinical trial meant to confirm previous trial findings supporting Zygel as a cannabidiol treatment in a specific subset of Fragile X syndrome patients. The new trial, called RECONNECT, is expected to launch before October, and will mainly enroll children and adolescents with a complete (100%) methylation of FMR1, the gene mutated in Fragile X.

Parkinson’s Therapy May Hold Promise for Fragile X

Fragile X News Today Borreca, Antonella • Martire, Alberto • Research Updates February 24, 2021AFQ056, drug tolerance, glutamate, mGluR5, Novartis

A study funded by FRAXA in Italy has encouraging results for people with Fragile X: drugs that block adenosine receptors (A2A) reversed signs of Fragile X in a mouse model.

“One of the most intriguing things about this study is that it points to an entire drug class (not just the one drug used) as potentially therapeutic for Fragile X. Many available compounds block A2A receptors, and we know they are safe and effective.

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Fragile X News Today Community • Treatment February 2, 2021December 9, 2021Adeno-associated virus, FDA, mRNAs

The two COVID-19 vaccines that recently received emergency approval from the US and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies.

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

Fragile X News Today Research Updates November 23, 2020November 22, 2021Alzheimer's, autism, drug tolerance, FDA, GABA, mGluR5, seizure

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, endocannabinoid, FDA, Zygel

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 Research Updates March 13, 2020November 22, 2021Alzheimer'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 Research Updates December 13, 2019November 22, 2021drug repurposing, Healx

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.