Fragile X News Today

May 26, 2020May 26, 2020

Blocking GSK Alpha Enzyme Safely Reverses Fragile X Symptoms in Mouse Model

Blocking an enzyme called GSK3 alpha seems to ease many symptoms of fragile X syndrome — including abnormal protein production, seizures induced by loud tones, learning and memory deficits, and excess ... Read more

May 19, 2020May 20, 2020

Trial Results for Zygel, CBD Gel Treatment for Fragile X, Likely by Mid-year

COVID-19 News, Fragile X News Today

Top-line results from the CONNECT-FX trial testing the cannabidiol (CBD) gel Zygel as a potential treatment for behavioral symptoms in children and teenagers with fragile X syndrome are expected by late June. ... Read more

May 12, 2020May 12, 2020

OV101 Well-tolerated, Effective in Fragile X Patients, Phase 2 Trial Shows

Fragile X News Today

Treatment with OV101 (gaboxadol) was well-tolerated and significantly eased behavioral and functional symptoms in people with fragile X syndrome, top-line data from a Phase 2 clinical trial show. The results support the ... Read more

May 5, 2020May 5, 2020

Lower Quality of Life Linked to Behavioral Problems in Fragile X Children, Parents Report

Fragile X News Today

Poorer health-related quality of life (HRQoL) correlate with greater emotional, behavioral, and school problems in children with fragile X syndrome, according to a study involving parent questionnaires. The study, “Examination ... Read more

April 29, 2020June 1, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

COVID-19 News, Fragile X News Today

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their ... Read more

April 29, 2020April 30, 2020

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Fragile X News Today

FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue ... Read more

April 28, 2020April 28, 2020

Expressive Language Sampling May Aid Research in Fragile X

Fragile X News Today

A procedure for measuring language skills, called expressive language sampling (ELS), could be used to assess treatment outcomes in people with fragile X syndrome and other conditions characterized by intellectual disability, ... Read more

April 21, 2020April 21, 2020

Clinical Symptoms and/or Family History of FXS Identify 96% of FXS Cases, Canadian Study Says

Fragile X News Today

Genetic testing for fragile X syndrome (FXS) should be reserved as a second-line strategy for people without clinical features or family history suggestive of FXS, according to a Canadian study. ... Read more

April 14, 2020April 14, 2020

Antipsychotic Medication Trifluoperazine Shows Promise for Fragile X, Study Finds

Fragile X News Today

Trifluoperazine, an approved antipsychotic medication normally prescribed to treat schizophrenia and anxiety, may be a promising therapeutic candidate for people with fragile X syndrome, a new study suggests. The study, ... Read more

April 7, 2020April 7, 2020

Urine microRNA Is Potential Biomarker for Fragile X, Early Study Suggests

Fragile X News Today

A small RNA molecule detectable in urine, called miR-125a, may provide a new biomarker for fragile X syndrome that could be used to group patients based on individual differences in ... Read more

April 2, 2020May 10, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

COVID-19 News, Fragile X News Today

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

March 31, 2020May 8, 2020

Israeli Clinic for Fragile X, One of World’s Largest, Adapting with COVID-19

COVID-19 News, Fragile X News Today

Caring for kids with fragile X syndrome — the most common inherited cause of intellectual disability — is challenging even under normal circumstances. Now, as the worst pandemic in more ... Read more

March 27, 2020May 8, 2020

NORD Partners with ‘Sing Me a Story’ for July 18-20 Living Rare Forum

COVID-19 News, Fragile X News Today

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The ... Read more

March 24, 2020March 24, 2020

Problems with Complex Speech, Talkativeness Seen as Common to Fragile X

Fragile X News Today

A tool to assess language, called expressive language sampling (ELS), found that people with fragile X syndrome have less complex speech, more disruptions in speech, greater talkativeness, and a lesser ... Read more

March 19, 2020March 21, 2020

Independence of Fragile X Patients Increases Until Their 30s, Then Starts to Decline, Study Says

Fragile X News Today

People with fragile X syndrome experience greater independence in daily activities and less severe behavioral problems over time, a trend that stabilizes or reverses when they reach their 30s, a ... Read more