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March 19, 2020March 19, 2020

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

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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

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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

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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

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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

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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

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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

March 3, 2020March 3, 2020

FDA Clears 1st Genetic Test for Fragile X Patients and Carriers

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The U.S. Food and Drug Administration (FDA) has authorized the marketing of Asuragen‘s AmplideX Fragile X Dx and Carrier Screen Kit, a first genetic test to help diagnose fragile ... Read more

March 2, 2020March 2, 2020

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

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Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, ... Read more

February 27, 2020February 27, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

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Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. ... Read more

February 20, 2020March 3, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

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An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

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