Updates

April 18, 2019April 18, 2019
CRISPR-Gold, Using Non-viral Carrier, Edits Fragile X Gene in Mice to Ease Exaggerated Behaviors

A newly developed version of the gene editing technology CRISPR — a non-viral delivery system called CRISPR-Gold — can effectively edit an autism-associated gene ... Read more
April 16, 2019April 16, 2019
Mindfulness Program Run on App May Help Ease Stress in Women with Fragile X Children, Study Says

A mindfulness program run on a free mobile app may be a feasible way of easing stress in mothers of children with fragile X ... Read more
April 11, 2019April 11, 2019
Telehealth Is Feasible, Effective Way to Treat Behavioral Issues in FRX, Study Shows

Implementing behavioral treatment via telehealth is a feasible and potentially cost-effective strategy for treating behavioral problems in boys with fragile X syndrome, a study ... Read more
April 9, 2019April 9, 2019
Patient’s Age, Family Income are Factors for Accessing Healthcare, FXS Caregivers Survey Reveals

Caregivers of younger children with fragile X syndrome (FXS), and those with lower family incomes, report more difficulties in accessing specialized healthcare in the ... Read more
April 4, 2019April 4, 2019
Green Tea Compound Significantly Improves Cognitive Function in Fragile X Patients, Study Finds

Treatment with a compound found in green tea leaves significantly improved cognitive function in patients with fragile X syndrome, a study shows. This new ... Read more

News

Early Social Avoidance Can Predict Autism but Not Anxiety in Boys with FXS, Study Says
April 2, 2019April 2, 2019
Increased social avoidance during infancy and early childhood in boys with fragile X syndrome (FXS) is ...
News

Pediatricians Unfamiliar With Guidelines for Genetic Testing of Children With Global Developmental Delay, Study Finds
March 28, 2019March 28, 2019
Pediatricians might not be aware of guidelines for genetic testing in children with global ...
News

Mavoglurant Rescues Specific Neural Networks in FXS Mice, Study Finds
March 26, 2019March 26, 2019
Mavoglurant (AFQ056), a candidate for the treatment of fragile X syndrome (FXS), restored functionality ...
News

Qualitative Study Identifies Major Concerns of Young Adults With Fragile X and Their Parents
March 22, 2019March 22, 2019
Young adults with fragile X syndrome (FXS) and their parents have identified several areas of ...
News

Combo Treatment Can Reactivate FMR1 Gene in Fragile X, Study Shows
March 19, 2019March 19, 2019
A combination of two chemical modulators of gene activity can stably reactivate the FMR1 gene, ...
News

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 14, 2019
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will ...

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Decisional Capacity of Women With Fragile X Surpasses That of Men, Study Says
March 12, 2019March 12, 2019
Women with fragile X syndrome surpass men in their decisional capacity for informed consent about ...
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 12, 2019March 11, 2019
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ...
Current FXTAS Diagnostic Criteria Show Low Accuracy, Study Finds
March 7, 2019March 7, 2019
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 5, 2019March 5, 2019
Zynerba Pharmaceuticals Receives U.S. Patent for Cannabidiol Gel ZYN002
March 5, 2019March 5, 2019
Understanding of How Females Inherit Traits May Shed Light on Fragile X, Rett Syndromes, Study Says
February 28, 2019February 28, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 26, 2019February 25, 2019
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ...
Vienna to Host RARE2019 Meeting on Rare Diseases
February 21, 2019February 20, 2019
Information, Support May Persuade Parents of FXS Children to Join Trials
February 19, 2019February 19, 2019
Defective Mitochondria Could Explain Neurological Impairment in Fragile X, Research Shows
February 14, 2019February 14, 2019

Mainstreaming Benefits Girls with Fragile X Outside Classroom, Study Suggests
February 12, 2019February 12, 2019
Students with fragile X who spend more time in regular classrooms are more likely to ...
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 11, 2019
Language Use Differs Between Boys with Both Autism and FXS and Autism Alone, Study Shows
February 8, 2019February 8, 2019

News

FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says
February 1, 2019February 1, 2019
Using eye-tracking technologies, researchers have shown that patients with fragile X syndrome avoid direct eye ...
News

Anxiety, Learning and Behavior Issues Top Concerns in Fragile X Syndrome, Survey Shows
January 29, 2019January 29, 2019
Anxiety, learning difficulties, and behavior problems are the main areas of concern in patients ...
News

Novartis’ Mavoglurant May Improve Visual Attention, Pupil Reactivity in Fragile X, Study Says
January 24, 2019January 24, 2019
Adolescents and adults diagnosed with fragile X syndrome (FXS) showed improvement in social gaze ...
News

Inhibiting HDAC6 Enzyme Has Potential for Treating FRX, Mouse Study Finds
January 22, 2019January 22, 2019
Inhibiting the HDAC6 enzyme with an experimental agent called SW-100 improved memory and lowered ...
News

Effort Afoot to Expand ‘Early Check’ Newborn Screening for Fragile X in the U.S.
January 15, 2019January 15, 2019
Early Check, a free newborn screening program for fragile X syndrome (FXS) that recently began testing in ...
News

Tetra Discovery and Shionogi Team Up to Further Study BPN14770 for Potential in Fragile X, Other Disorders
January 8, 2019January 8, 2019
A new collaboration between Tetra Discovery Partners and Shionogi & Co. is aimed at ...

March 6, 2018March 6, 2018

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

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