Urine microRNA Is Potential Biomarker for Fragile X, Early Study Suggests
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 ... Read more
Updates
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NORD’s Advice on COVID-19: You’re Stronger Than You Think
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 ... Read more -
Israeli Clinic for Fragile X, One of World’s Largest, Adapting with COVID-19
Caring for kids with fragile X syndrome — the most common inherited cause of intellectual disability — is challenging even under normal circumstances. Now, ... Read more -
NORD Partners with ‘Sing Me a Story’ for July 18-20 Living Rare Forum
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 ... Read more -
Problems with Complex Speech, Talkativeness Seen as Common to Fragile X
A tool to assess language, called expressive language sampling (ELS), found that people with fragile X syndrome have less complex speech, more disruptions in ... Read more
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Independence of Fragile X Patients Increases Until Their 30s, Then Starts to Decline, Study Says
People with fragile X syndrome experience greater independence in daily activities and less severe ... -
New Way of Testing for Fragile X May Be Faster and Cheaper
Using a genetic technique called triplet-primed polymerase chain reaction (TP-PCR) could enable practical and ... -
Companies Move to Advance Potential Cognitive Treatment for Fragile X
Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment ... -
CONNECT-FX Trial Testing Zygel Completes Enrollment of Young Fragile X Patients
Zynerba Pharmaceuticals has completed enrollment for the CONNECT-FX trial testing its cannabidiol gel, Zygel, ... -
Zygel May Ease Fragile X Effect on Children’s Lives, Trial Data Suggest
Treatment with Zygel (ZYN002) may help ease the significant effects fragile X syndrome (FXS) has ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ...
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Rapamycin Treatment Failed to Improve Fragile X Behavioral Symptoms in Mouse Study
News
An older existing medication that targets a pathway thought to be involved in fragile X syndrome failed to reverse any behavioral characteristics in mouse models of the disease, and was found to further compromise social skills in the mice. ... Read more