Fragile X News Today

January 15, 2021January 15, 2021

National Fragile X Foundation Leading Patient-Focused Drug Development Meeting

The National Fragile X Foundation (NFXF) will host a virtual patient-focused drug development (PFDD) meeting for fragile X syndrome on March 3. To be held from 10 a.m. to 2 p.m. ... Read more

January 12, 2021January 13, 2021

NIH Grant Boosts Research Into Faulty Protein Production in Fragile X

Fragile X News Today

A nearly $2 million grant from the National Institute of Mental Health, part of the National Institutes of Health (NIH), will help researchers understand the mechanisms underlying impaired protein production ... Read more

January 5, 2021January 7, 2021

Telehealth Can Expand Access to Behavioral Treatments for Fragile X

Fragile X News Today

Telehealth, or telemedicine, can expand access to behavioral treatments for children with fragile X syndrome, a clinical study suggests. An approach called functional communication training, or FCT, delivered via telehealth, ... Read more

December 22, 2020December 22, 2020

Study Underscores Critical Role of FMRP in Brain Development

Fragile X News Today

A study in mice has identified 865 gene targets of the fragile X mental retardation protein (FMRP) — which is missing in people with fragile X syndrome — including more ... Read more

December 18, 2020December 18, 2020

Zygel, CBD Gel, to Enter Pivotal Trial in Patients With Severe Symptoms

Fragile X News Today

Zynerba Pharmaceuticals announced plans to open a pivotal clinical trial next year for its cannabidiol (CBD) gel Zygel in fragile X syndrome patients with severe symptoms, a group that showed improvements in ... Read more

December 11, 2020December 11, 2020

DNA Marks May Help Diagnose, Predict Prognosis in Fragile X, Study Suggests

Fragile X News Today

Techniques that evaluate the pattern of DNA methylation — a chemical modification normally associated with gene silencing — in a portion of the FMR1 gene are feasible and highly sensitive ... Read more

December 8, 2020December 9, 2020

Confluence Granted $100,000 to Advance Fragile X Social, Communication Therapy

Fragile X News Today

Confluence Pharmaceuticals will use a $100,000 investment to further its development of ACP, an investigational therapy that aims to treat social and communication impairments related to fragile X syndrome and autism ... Read more

December 4, 2020December 5, 2020

International Fragile X Premutation Registry Launches in US

Fragile X News Today

The National Fragile X Foundation (NFXF) has launched the International Fragile X Premutation Registry to assist and encourage research into conditions caused by so-called premutations in the gene associated with fragile ... Read more

December 1, 2020December 1, 2020

Experimental Fragile X Treatment Arbaclofen Back in Development

Fragile X News Today

Allos Pharma has obtained exclusive rights to arbaclofen, a potential treatment for fragile X syndrome. The news comes more than seven years after Seaside Therapeutics decided to stop a clinical ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

COVID-19 News, Fragile X News Today

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 24, 2020November 24, 2020

Changes in Brain Circuits May Underlie Fragile X Sound Sensitivity, Study Suggests

Fragile X News Today

Scientists found that small fish larvae lacking a functional fmr1 gene — the fish equivalent to the human FMR1 gene that, when mutated, causes fragile X syndrome — show changes ... Read more

November 17, 2020November 17, 2020

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

Fragile X News Today

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 ... Read more

November 13, 2020November 14, 2020

Study Seeking to Link Brain Processes With Cognition, Behavior Now Recruiting

Fragile X News Today

A Phase 1 clinical trial is recruiting adults with fragile X syndrome to study the brain processes underlying altered behavior and cognition, according to the FRAXA Research Foundation. Conducted at ... Read more

November 6, 2020November 6, 2020

Fragile X Protein May Be Crucial for Cells That Support Neurons, Study Finds

Fragile X News Today

Loss of the fragile X mental retardation protein (FMRP), the protein missing in people with fragile X syndrome, was found in mouse and human astroglia — nerve cells that support and ... Read more

November 3, 2020November 3, 2020

BPN14770 Shows Cognition Benefits in Phase 2 Clinical Trial

Fragile X News Today

The investigational treatment BPN14770 improved language skills, cognition, and caregiver-assessed daily functioning ability in adults with fragile X syndrome, top-line results of a Phase 2 clinical trial show. “We are ... Read more