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January 16, 2020January 16, 2020

Mothers of Fragile X Children Have Cognitive Problems Linked to Their Genetic Profile and Older Age, Study Finds

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Specific cognitive deficits in mothers of children with fragile X syndrome are significantly linked to their genetic profile and are worse in older age, a study has found. The ... Read more

January 14, 2020January 14, 2020

CONNECT-FX Trial Testing Cannabidiol Gel Zygel in Fragile X Close to Complete Enrollment, Zynerba Announces

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The CONNECT-FX trial testing the cannabidiol (CBD) gel Zygel as a treatment for behavioral symptoms in children and teenagers with fragile X syndrome has nearly completed enrollment. Zynerba, the ... Read more

January 9, 2020January 9, 2020

Results of Phase 2 Trial Testing OV101 in Fragile X Expected Later This Year

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Ovid Therapeutics is expecting to have data from a Phase 2 trial testing OV101 (gaboxadol) in teenagers and young adults with fragile X syndrome later this year. The main goal of ... Read more

January 7, 2020January 7, 2020

ICAM5 Protein May Be Target to Treat Cognitive Impairment in Fragile X, Study Suggests

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Reducing the level of a protein called ICAM5 in the brain improved memory and eased anxiety in a mouse model of fragile X syndrome, a new study shows. The ... Read more

January 2, 2020January 2, 2020

Several Proteins Tied to Neuronal Defects May Offer Way of Treating FXTAS, Study Suggests

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A group of proteins linked with nerve cell defects could represent promising treatment targets for people with fragile X tremor-ataxia syndrome (FXTAS), a study in mice suggested. The study, “Reduction ... Read more

December 19, 2019December 19, 2019

Abnormal Gaze Linked to Social Cognition in Women With FMR1 Premutation, Study Finds

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Atypical gazing when looking at facial expressions is associated with social cognition and language abilities in women with a premutation in the FMR1 gene, a new study has found. ... Read more

December 17, 2019December 17, 2019

Hallmark DNA Repeats Not Tied to Typical Gene Silencing in Fragile X Syndrome, Case Study Suggests

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The expansion of DNA repeats in the FMR1 gene, which leads to fragile X syndrome, is independent of changes that stop production of the resulting FMRP protein, according to ... Read more

December 12, 2019December 12, 2019

New Partners Aim to Accelerate the Discovery and Repurposing of Medicines for Rare Neurological Diseases

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Healx and Boehringer Ingelheim are partnering to speed up the discovery of new treatments for rare neurological diseases such as fragile X syndrome. Under the agreement, Healx’s artificial intelligence ... Read more

December 11, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 10, 2019December 10, 2019

New Method of Analyzing DNA Repeats May Help Diagnose Fragile X and Other Disorders, Study Reports

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A new method can accurately quantify genetic variants called short tandem repeats (STRs) and may improve diagnosis of fragile X syndrome and other disorders, a study suggests. The method ... Read more

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