Updates

June 18, 2019June 18, 2019
Social Avoidance Behaviors Emerge in Infancy, Increase During Childhood, Study Finds

Social avoidance, a common behavior among boys with fragile X syndrome, emerges during infancy, increases in severity across childhood, and stabilizes through adolescence and ... Read more
June 18, 2019June 17, 2019
‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those ... Read more
June 11, 2019June 11, 2019
FXTAS Should be Considered in the Diagnosis of Patients With Essential Tremor Who Show Certain Signs, Study Says

Fragile X-associated tremor/ataxia syndrome (FXTAS) should be considered in the diagnosis of patients with essential tremor who show signs of cerebellar involvement and/or drug-induced movement ... Read more
June 6, 2019June 6, 2019
Early Short-term Lovastatin Treatment May Correct Learning Deficits in Fragile X Syndrome, Rat Study Suggests

Short-term treatment early in life with lovastatin, a medication widely prescribed for lowering cholesterol, can rescue memory deficits in a rat model of fragile ... Read more
May 30, 2019May 30, 2019
Self-Injury and Aggressive Behaviors Highly Persistent Among Males, Longitudinal Study Says

Self-injurious and aggressive behaviors are highly prevalent and persistent among males with fragile X syndrome (FXS), a study found. These results may have implications ... Read more

News

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
May 28, 2019May 24, 2019
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims ...
News

Diabetes Medicine Metformin May Improve Cognition, Behavior Symptoms in Fragile X Syndrome Patients, Case Study Reports
May 23, 2019May 23, 2019
One year of treatment with approved oral diabetes medicine, metformin, helped to improve the ...
News

Modulation of Potassium Channels May Treat Symptoms in FXS, Study Finds
May 21, 2019May 21, 2019
Compounds that can restore potassium balance in some brain regions may potentially treat sensory ...
News

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
May 16, 2019May 15, 2019
With 250 rare diseases newly identified every year, scientists can barely keep up — ...
News

Cannabidiol Gel ZYN002 Granted Fast Track Status for Treatment of Behavioral Symptoms in Fragile X Syndrome
May 7, 2019May 7, 2019
Zygel (ZYN002 CBD gel) has been granted fast track designation by the United States’ ...
News

Confluence Gets $200K from University-affiliated Venture Fund to Develop Fragile X Treatment
April 30, 2019April 30, 2019
An Indiana University-affiliated venture fund has invested $200,000 in Confluence Pharmaceuticals to continue development ...

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Cognitive Training Program Provides Benefits for Young Fragile X Syndrome Patients, Trial Finds
April 23, 2019April 23, 2019
A computer-based cognitive training program called Cogmed can improve working memory and cognition and ...
CRISPR-Gold, Using Non-viral Carrier, Edits Fragile X Gene in Mice to Ease Exaggerated Behaviors
April 18, 2019April 18, 2019
A newly developed version of the gene editing technology CRISPR — a non-viral delivery ...
Mindfulness Program Run on App May Help Ease Stress in Women with Fragile X Children, Study Says
April 16, 2019April 16, 2019
Telehealth Is Feasible, Effective Way to Treat Behavioral Issues in FRX, Study Shows
April 11, 2019April 11, 2019
Patient’s Age, Family Income are Factors for Accessing Healthcare, FXS Caregivers Survey Reveals
April 9, 2019April 9, 2019
Green Tea Compound Significantly Improves Cognitive Function in Fragile X Patients, Study Finds
April 4, 2019April 4, 2019

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 ...
Pediatricians Unfamiliar With Guidelines for Genetic Testing of Children With Global Developmental Delay, Study Finds
March 28, 2019March 28, 2019
Mavoglurant Rescues Specific Neural Networks in FXS Mice, Study Finds
March 26, 2019March 26, 2019
Qualitative Study Identifies Major Concerns of Young Adults With Fragile X and Their Parents
March 22, 2019March 22, 2019

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, ...
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 14, 2019
Decisional Capacity of Women With Fragile X Surpasses That of Men, Study Says
March 12, 2019March 12, 2019

News

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 ...
News

Current FXTAS Diagnostic Criteria Show Low Accuracy, Study Finds
March 7, 2019March 7, 2019
Given the genetic variability behind the disease, current clinical diagnostic criteria for fragile X-linked ...
News

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 5, 2019March 5, 2019
Rare diseases affect about 30 million Americans — roughly the same number as those ...
News

Zynerba Pharmaceuticals Receives U.S. Patent for Cannabidiol Gel ZYN002
March 5, 2019March 5, 2019
Zynerba Pharmaceuticals’ cannabidiol gel ZYN002 has received a U.S. patent for the treatment of ...
News

Understanding of How Females Inherit Traits May Shed Light on Fragile X, Rett Syndromes, Study Says
February 28, 2019February 28, 2019
New understanding of the genetic mechanisms involved in female inheritance and X chromosome silencing ...
News

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 ...

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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