Updates

November 24, 2020November 24, 2020
Changes in Brain Circuits May Underlie Fragile X Sound Sensitivity, Study Suggests

Scientists found that small fish larvae lacking a functional fmr1 gene — the fish equivalent to the human FMR1 gene that, when mutated, causes ... Read more
November 20, 2020November 20, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and ... Read more
November 17, 2020November 17, 2020
Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model

Long-term, but not short-term, treatment with bryostatin-1 — Neurotrope’s lead investigational therapy — arrested such behavioral and cognitive symptoms as hyperactivity, difficulties with daily ... Read more
November 13, 2020November 13, 2020
Study Seeking to Link Brain Processes With Cognition, Behavior Now Recruiting

A Phase 1 clinical trial is recruiting adults with fragile X syndrome to study the brain processes underlying altered behavior and cognition, according to ... Read more
November 6, 2020November 6, 2020
Fragile X Protein May Be Crucial for Cells That Support Neurons, Study Finds

Loss of the fragile X mental retardation protein (FMRP), the protein missing in people with fragile X syndrome, was found in mouse and human astroglia ... 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

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

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

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

UC Davis Establishing 4-Year Program for Students With Fragile X, Other Disabilities

Patrick Wild Centre Opens Website for Its Fragile X Registry

Connecta Therapeutics Raises Funds to Advance Potential Fragile X Therapy

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

$25M NIH Grant to Support Fragile X Research at 3 Centers

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Strong Placebo Effect Warrants Better Trial Design in Fragile X, Study Finds

Hospitalization After Emergency Department Visit Common in Fragile X Patients, Study Finds

Zynerba, FDA to Discuss Next Steps for Zygel in Fragile X Treatment

Closing Leaky Mitochondria Halts Behavioral Problems in Fragile X, Study Suggests

Newly Discovered Mechanisms Controlling Memory, Learning Could Lead to Therapies

Same But Different Photo Contest Celebrates People With Rare Diseases

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Better Communication Needed to Help Fragile X Patients Transition Into Adulthood, Study Suggests

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Fewer ‘Sensing Antennas’ in Brain Nerve Cells May Contribute to Fragile X, Mouse Study Asserts

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

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

Subscribe to our mailing list

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

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

UC Davis Establishing 4-Year Program for Students With Fragile X, Other Disabilities

Patrick Wild Centre Opens Website for Its Fragile X Registry

Connecta Therapeutics Raises Funds to Advance Potential Fragile X Therapy

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

$25M NIH Grant to Support Fragile X Research at 3 Centers

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Fragile X Girls Less Aware of Bullying, Study Suggests

Strong Placebo Effect Warrants Better Trial Design in Fragile X, Study Finds

Hospitalization After Emergency Department Visit Common in Fragile X Patients, Study Finds

Zynerba, FDA to Discuss Next Steps for Zygel in Fragile X Treatment

Closing Leaky Mitochondria Halts Behavioral Problems in Fragile X, Study Suggests

Newly Discovered Mechanisms Controlling Memory, Learning Could Lead to Therapies

Same But Different Photo Contest Celebrates People With Rare Diseases

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Better Communication Needed to Help Fragile X Patients Transition Into Adulthood, Study Suggests

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Fewer ‘Sensing Antennas’ in Brain Nerve Cells May Contribute to Fragile X, Mouse Study Asserts