News

April 2, 2020April 6, 2020

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

News, syndicated

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 ... Read more

March 31, 2020March 31, 2020

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

News

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 ... Read more

March 27, 2020March 27, 2020

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

News

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. ... Read more

March 24, 2020March 24, 2020

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

News

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 ... Read more

March 19, 2020March 19, 2020

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

News

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, ... Read more

March 17, 2020March 17, 2020

New Way of Testing for Fragile X May Be Faster and Cheaper

News

Using a genetic technique called triplet-primed polymerase chain reaction (TP-PCR) could enable practical and inexpensive diagnosis of fragile X syndrome, a study suggests. The study, “Repeat expansion and methylation-sensitive ... Read more

March 12, 2020March 12, 2020

Companies Move to Advance Potential Cognitive Treatment for Fragile X

News

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. ... Read more

March 10, 2020March 10, 2020

CONNECT-FX Trial Testing Zygel Completes Enrollment of Young Fragile X Patients

News

Zynerba Pharmaceuticals has completed enrollment for the CONNECT-FX trial testing its cannabidiol gel, Zygel, as a treatment for behavioral symptoms in children and teenagers with fragile X syndrome. Recruitment ... Read more

March 6, 2020March 6, 2020

Zygel May Ease Fragile X Effect on Children’s Lives, Trial Data Suggest

News

Treatment with Zygel (ZYN002) may help ease the significant effects fragile X syndrome (FXS) has on children, updated results of a Phase 1/2 clinical trial suggest. Zynerba Pharmaceuticals presented the ... Read more

March 5, 2020March 6, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News

In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

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