Updates

January 28, 2021January 28, 2021
COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to ... Read more
January 26, 2021January 26, 2021
Anticonvulsant Shows Promise at Treating Fragile X in Mouse Model

The anticonvulsant carbamazepine can normalize behavior and memory in a mouse model of fragile X syndrome (FXS), suggesting that this medication might be re-purposed ... Read more
January 22, 2021January 22, 2021
Lower Brain Levels of mGluR5 Receptor Found in Fragile X Men

Brain scans in men with fragile X syndrome showed reduced levels of the mGluR5 receptor — a protein receptor known to help regulate nerve ... Read more
January 20, 2021January 20, 2021
Art Contest Salutes Creativity of Rare Disease Communities

Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare ... Read more
January 19, 2021January 20, 2021
NORD’s Caregiver Respite Program Continues Through Pandemic

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet ... Read more

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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

Updates

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Anticonvulsant Shows Promise at Treating Fragile X in Mouse Model

Lower Brain Levels of mGluR5 Receptor Found in Fragile X Men

Art Contest Salutes Creativity of Rare Disease Communities

NORD’s Caregiver Respite Program Continues Through Pandemic

National Fragile X Foundation Leading Patient-Focused Drug Development Meeting

NIH Grant Boosts Research Into Faulty Protein Production in Fragile X

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Telehealth Can Expand Access to Behavioral Treatments for Fragile X

Study Underscores Critical Role of FMRP in Brain Development

Zygel, CBD Gel, to Enter Pivotal Trial in Patients With Severe Symptoms

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International Fragile X Premutation Registry Launches in US

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Experimental Fragile X Treatment Arbaclofen Back in Development

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Changes in Brain Circuits May Underlie Fragile X Sound Sensitivity, Study Suggests

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model

Study Seeking to Link Brain Processes With Cognition, Behavior Now Recruiting

Fragile X Protein May Be Crucial for Cells That Support Neurons, Study Finds

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

BPN14770 Shows Cognition Benefits in Phase 2 Clinical Trial

Screening for Fragile X Carriers May Be Cost-free Option for Couples in Australia

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

FMRP Protein Helps Prevent DNA Damage, Maintain Genome Stability, Study Finds

Phase 2 Trial Planned for Xanamem, Oral Treatment for Fragile X

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest

Rapamycin Treatment Failed to Improve Fragile X Behavioral Symptoms in Mouse Study

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Anticonvulsant Shows Promise at Treating Fragile X in Mouse Model

Lower Brain Levels of mGluR5 Receptor Found in Fragile X Men

Art Contest Salutes Creativity of Rare Disease Communities

NORD’s Caregiver Respite Program Continues Through Pandemic

National Fragile X Foundation Leading Patient-Focused Drug Development Meeting

NIH Grant Boosts Research Into Faulty Protein Production in Fragile X

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Telehealth Can Expand Access to Behavioral Treatments for Fragile X

Study Underscores Critical Role of FMRP in Brain Development

Zygel, CBD Gel, to Enter Pivotal Trial in Patients With Severe Symptoms

Subscribe to our mailing list

DNA Marks May Help Diagnose, Predict Prognosis in Fragile X, Study Suggests

Confluence Granted $100,000 to Advance Fragile X Social, Communication Therapy

International Fragile X Premutation Registry Launches in US

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Experimental Fragile X Treatment Arbaclofen Back in Development

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Changes in Brain Circuits May Underlie Fragile X Sound Sensitivity, Study Suggests

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model

Study Seeking to Link Brain Processes With Cognition, Behavior Now Recruiting

Fragile X Protein May Be Crucial for Cells That Support Neurons, Study Finds

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

BPN14770 Shows Cognition Benefits in Phase 2 Clinical Trial

Screening for Fragile X Carriers May Be Cost-free Option for Couples in Australia

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

FMRP Protein Helps Prevent DNA Damage, Maintain Genome Stability, Study Finds

Phase 2 Trial Planned for Xanamem, Oral Treatment for Fragile X

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest