Fragile X News Today

June 18, 2021June 18, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 11, 2021June 11, 2021

Survey: COVID-19 Lockdowns Affected Sleep, Well-being of Fragile X Children

COVID-19 News, Fragile X News Today

COVID-19 lockdowns posed additional challenges for mothers and their children — from toddlers to teens — with fragile X syndrome, who saw their sleep habits and well-being disrupted, a small survey ... Read more

June 8, 2021June 8, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Fragile X News Today

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 4, 2021June 4, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Fragile X News Today

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 1, 2021June 2, 2021

Breast Milk for 1 Year for Fragile X Infants May Reduce Autism Risk

Fragile X News Today

Infants with fragile X syndrome who are fed with breast milk for a minimum of one year have a decreased prevalence of autism, a retrospective study found. However, breast milk ... Read more

May 28, 2021May 28, 2021

Study Will Measure Serotonin, Gut Microbes in ASDs

Fragile X News Today

Nova Mentis has launched an observational study examining the interconnected roles that gut microbes, serotonin, and the immune system play in the gut and brain development of people with autism spectrum ... Read more

May 25, 2021May 25, 2021

UC Davis Offers Program for Intellectually Disabled

Fragile X News Today

Applications are being accepted for an innovative new four-year college program at the University of California, Davis (UC Davis) for students with intellectual disabilities such as fragile X syndrome. The ... Read more

May 21, 2021May 21, 2021

Artificial Intelligence Tool May Help Speed Diagnosis

Fragile X News Today

An artificial intelligence (AI) model based only on the occurrence of health conditions previously and newly associated with fragile X syndrome accurately identified fragile X patients five years earlier than diagnosis, ... Read more

May 18, 2021May 18, 2021

Researchers Shed Light on Metabolic Profile of Fragile X Mouse Model

Fragile X News Today

A mouse model of fragile X syndrome has higher body weight and distinct metabolic changes relative to healthy mice and mouse models of two other neurodevelopmental disorders, a study shows. ... Read more

May 14, 2021May 15, 2021

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

Fragile X News Today

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 ... Read more

May 11, 2021May 11, 2021

Zygel Led to Meaningful Behavioral Improvements in Certain Youngsters

Fragile X News Today

Zynerba Pharmaceuticals’ experimental cannabidiol (CBD) gel Zygel results in statistically significant and clinically meaningful reductions in behavioral symptoms in children and adolescents with fragile X syndrome (FXS) and a fully methylated ... Read more

May 7, 2021May 7, 2021

New Autism Data Bank Will Help Scientists Study Serotonin Levels

Fragile X News Today

Nova Mentis is creating a North American data bank of people with autism spectrum disorders (ASD), which it will use for a study evaluating serotonin levels in ASD patients, including ... Read more

May 4, 2021May 4, 2021

BPN14770 Improved Men’s Language, Daily Functions

Fragile X News Today

Tetra Therapeutics’ investigational oral therapy BPN14770 safely and effectively improves cognitive function, particularly language-related domains, and daily functioning in men with fragile X syndrome, according to data from a Phase ... Read more

April 30, 2021April 30, 2021

Signaling Defects Affecting Nerve Cell Growth Identified in Fragile X

Fragile X News Today

Abnormal protein production associated with fragile X syndrome was coupled with altered cell growth that favored fast-growing cell types over those that become nerve cells during early development, a study ... Read more

April 27, 2021April 27, 2021

Better Info Access May Boost Life Quality for Men with Premutations

Fragile X News Today

Better access to information about fragile X-associated ataxia/tremor syndrome (FXTAS) could help improve the quality of life for men carrying fragile X syndrome premutations, according to the results of a ... Read more