Fragile X News Today

August 4, 2020August 4, 2020

NIH Grant Supports Research Into Potential Targets for Fragile X Therapies

Investigators at the Mercer University’s College of Pharmacy were awarded almost $425,000 in three-year funding to advance work into serotonin receptors as potential targets for preventing seizures in people with ... Read more

July 24, 2020July 24, 2020

Zygel Improves Caregiver Impressions of Behavioral Symptoms in Some Fragile X Children

Fragile X News Today

The experimental cannabidiol (CBD) gel Zygel eased behavioral problems in children with fragile X syndrome and benefited those with greater disease severity according to their caregivers, new clinical trial data show. The data ... Read more

July 21, 2020July 22, 2020

Blood Proteins May Help Measure Effectiveness of Metformin in Fragile X

Fragile X News Today

Measuring the levels of three blood biomarkers could help identify patients with fragile X syndrome who are likely to benefit from treatment with the diabetes medicine metformin, a study suggests. The study, ... Read more

July 17, 2020July 17, 2020

Metformin Seen to Help Correct Defects in Neurons Lacking FMRP Protein

Fragile X News Today

The common type 2 diabetes medication metformin eased the excessive production of proteins in nerve cells that were engineered in a lab to have the underlying defect seen in people with fragile ... Read more

July 14, 2020July 14, 2020

Understanding Changes in Synapses May Provide Therapeutic Clues for Fragile X, Mouse Study Suggests

Fragile X News Today

Scientists found evidence of a decoupling between structural and functional changes that weaken synapses (the place where nerve cells communicate). The findings may help identify new therapeutic targets and develop ... Read more

July 10, 2020July 10, 2020

National Fragile X Awareness Month Is Underway

Fragile X News Today

July is National Fragile X Awareness Month, an annual event set aside to bring attention to the rare genetic condition. Across the country, the fragile X syndrome community is observing ... Read more

July 7, 2020July 7, 2020

Zygel May Be Effective for Children with Severe Fragile X Symptoms, CONNECT-FX Trial Suggests

Fragile X News Today

Zynerba Pharmaceuticals‘ Zygel failed to significantly ease behavioral symptoms in the overall group of children with fragile X syndrome (FXS) who took part in the CONNECT-FX trial, top-line results show. ... Read more

June 23, 2020June 23, 2020

Parent Language Intervention Eases Speech, Social Impairments in Fragile X, Trial Finds

Fragile X News Today

A parent language intervention based on story-telling eases the severity of speech and social impairments in children with fragile X syndrome, a study shows. The study also found that adding lovastatin, ... Read more

June 22, 2020July 24, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

COVID-19 News, Fragile X News Today

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support ... Read more

June 19, 2020June 19, 2020

Inflammation Not a Hallmark of Fragile X

Fragile X News Today

Low levels of pro-inflammatory proteins in the blood suggest that inflammation is not a hallmark of fragile X syndrome, according to a new study. The findings also indicated that assessing the levels ... Read more

June 12, 2020June 13, 2020

Trofinetide Safe and Effective at Higher Dose for Fragile X, Phase 2 Trial Finds

Fragile X News Today

Oral treatment with trofinetide was well tolerated and, at a higher dose, eased social avoidance, anxiety, hyperactivity and other symptoms of fragile X syndrome in adolescent and adult males, results ... Read more

June 10, 2020July 11, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

COVID-19 News, Fragile X News Today

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for ... Read more

June 9, 2020June 10, 2020

Genetic Screening Study Starts Recruiting Prospective Parents in Australia

COVID-19 News, Fragile X News Today

An Australian project is recruiting couples planning to have children or experiencing early pregnancy for a nationwide genetic study to screen for about 750 genetic conditions, including fragile X syndrome. ... Read more

June 5, 2020June 5, 2020

Specific Piece of FMRP Protein Eases Hyperactivity in Mouse Model

Fragile X News Today

Providing a fragment of the FMRP protein, which is missing in people with fragile X syndrome, eased hyperactivity and restored nerve cell communication in the brain, a study in a ... Read more

June 2, 2020June 3, 2020

Shionogi Acquires Tetra Therapeutics, Rights to Fragile X Treatment Candidate

Fragile X News Today

Shionogi has acquired Tetra Therapeutics, the developer of a treatment candidate known as BPN14770 for fragile X syndrome and Alzheimer’s disease. With the agreement, Shionogi obtains global rights to Tetra’s entire pipeline. It will continue ... Read more