Updates

February 14, 2019February 14, 2019
Defective Mitochondria Could Explain Neurological Impairment in Fragile X, Research Shows

Mutations in the FMR1 gene — the underlying cause of fragile X syndrome — result in mitochondria malformations inside developing brain nerve cells. Subsequently, this does ... Read more
February 12, 2019February 12, 2019
Mainstreaming Benefits Girls with Fragile X Outside Classroom, Study Suggests

Students with fragile X who spend more time in regular classrooms are more likely to perform functional tasks without help, a new study proposes. These ... Read more
February 11, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
February 8, 2019February 8, 2019
Language Use Differs Between Boys with Both Autism and FXS and Autism Alone, Study Shows

Adolescent autistic boys who also have fragile X syndrome converse differently when it comes to certain aspects of language than autistic boys without the disease, ... Read more
February 1, 2019February 1, 2019
FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says

Using eye-tracking technologies, researchers have shown that patients with fragile X syndrome avoid direct eye contact and show signs of social deficits linked to social ... Read more

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 ...
News

FMR1 Mutations Linked to Early Atypical Temperament, Study Shows
January 3, 2019January 3, 2019
Mutations in the FMR1 gene are associated with atypical temperament that can emerge as ...

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Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 21, 2018December 20, 2018
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures ...
Cannabidiol Gel ZYN002 Shows Sustained Benefits in Children and Teens with Fragile X, Zynerba Reports
December 18, 2018December 18, 2018
One year of treatment with the cannabidiol gel ZYN002, being developed by Zynerba Pharmaceuticals, can ...
Ability to Form Words, Sentences Linked with Autism Spectrum Disorder Severity in Young Males with Fragile X
December 5, 2018December 5, 2018
Mavoglurant Safe, Well-tolerated Long-term in Adolescents and Adults with Fragile X Syndrome, Studies Show
November 27, 2018November 27, 2018
World’s Largest Newborn Screening Study to Include Fragile X Syndrome
November 20, 2018November 20, 2018
Exposure to Pesticides Might Increase Severity of Fragile X Spectrum Disorders, Study Finds
November 13, 2018November 13, 2018

Cognitive Function Affects Gait, Motor Function in FXTAS, Study Finds
November 6, 2018November 6, 2018
Lower cognitive function affects gait and functional mobility in those with fragile X-associated tremor/ataxia ...
Fragile X Added to Newborn Screening in North Carolina Through New Study
October 30, 2018October 30, 2018
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 29, 2018October 29, 2018
Changes in Calcium Dynamics Within Nerve Cells May Be Biomarker for Fragile X, Study Reports
October 25, 2018October 26, 2018

Social Anxiety Traits Overlap Between Fragile X Syndrome, Autism Spectrum Disorder
October 23, 2018October 23, 2018
Social anxiety and autistic traits are prevalent in males with fragile X syndrome and ...
Therapeutics Targeting cAMP Signaling Can Help Improve Sensory Perception in Patients with Fragile X Syndrome
October 18, 2018October 18, 2018
Genome Folded Incorrectly in Fragile X Syndrome, Other Neurological Conditions, Study Finds
October 11, 2018October 11, 2018

News

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018October 9, 2018
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced ...
News

Irregular Visual Perception in a Mouse Model of Fragile X Syndrome Could be Treated using Designer Therapies
October 9, 2018October 9, 2018
Visual perception impairments in a mouse model of fragile X syndrome can be therapeutically ...
News

Simvastatin Does Not Show Same Brain Benefits for Fragile X as Lovastatin, Study Reports
October 4, 2018October 4, 2018
Lovastatin, a medication widely prescribed for lowering cholesterol, was recently discovered to correct brain ...
News

Allopregnanolone May Help Improve Cognitive Function in Fragile X-Associated Tremor/Ataxia Syndrome, Study Suggests
October 2, 2018October 2, 2018
Weekly infusions with allopregnanolone, a natural brain steroid, for up to 12 weeks may ...
News

Cellular ‘Cleaning System’ a Possible Therapeutic Target for Fragile X, Mouse Study Finds
September 27, 2018September 27, 2018
Autophagy, a natural process that acts as a “cleaning system” for cells, plays an ...
News

Researchers Identify Potential New Fragile X Therapeutic Target Called Kv1.2
September 25, 2018September 25, 2018
Researchers have found that increasing the activity of a specific potassium channel called Kv1.2 ...

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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