News

January 3, 2019January 3, 2019

FMR1 Mutations Linked to Early Atypical Temperament, Study Shows

News

Mutations in the FMR1 gene are associated with atypical temperament that can emerge as early as infancy, particularly among infants with fragile X syndrome, a study has shown. The ... Read more

December 21, 2018December 20, 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

December 18, 2018December 18, 2018

Cannabidiol Gel ZYN002 Shows Sustained Benefits in Children and Teens with Fragile X, Zynerba Reports

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One year of treatment with the cannabidiol gel ZYN002, being developed by Zynerba Pharmaceuticals, can provide sustained benefit to young patients with fragile X syndrome, data from a Phase 2 ... Read more

December 5, 2018December 5, 2018

Ability to Form Words, Sentences Linked with Autism Spectrum Disorder Severity in Young Males with Fragile X

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A greater ability to generate words, phrases and sentences correlates with lower autism spectrum disorder (ASD) symptom severity in adolescent and young adult males with fragile X syndrome (FXS), according ... Read more

November 27, 2018November 27, 2018

Mavoglurant Safe, Well-tolerated Long-term in Adolescents and Adults with Fragile X Syndrome, Studies Show

News

Mavoglurant is safe and well-tolerated in the long-term to treat adolescents and adults who have fragile X symptoms. The investigational compound also is associated with positive efficacy results, according ... Read more

November 20, 2018November 20, 2018

World’s Largest Newborn Screening Study to Include Fragile X Syndrome

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Fragile X syndrome is among the four rare genetic disorders to be included in the world’s largest newborn screening study for rare diseases. The screening, financially supported by the Angelman ... Read more

November 13, 2018November 13, 2018

Exposure to Pesticides Might Increase Severity of Fragile X Spectrum Disorders, Study Finds

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The frequency and severity of symptoms associated with fragile X spectrum disorders in the rural town of Ricaurte in Colombia are much higher compared to those of the general ... Read more

November 6, 2018November 6, 2018

Cognitive Function Affects Gait, Motor Function in FXTAS, Study Finds

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Lower cognitive function affects gait and functional mobility in those with fragile X-associated tremor/ataxia syndrome (FXTAS), a study has found. Linking quantitative gait assessment with neuropsychological tests may help ... Read more

October 30, 2018October 30, 2018

Fragile X Added to Newborn Screening in North Carolina Through New Study

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Fragile X syndrome testing will now be available for newborns in North Carolina by way of a new study led by RTI International to research the disease’s prevalence and the ... Read more

October 29, 2018October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

News

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit ... Read more

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