Updates

October 23, 2020October 23, 2020
Phase 2 Trial Planned for Xanamem, Oral Treatment for Fragile X

Actinogen Medical announced plans to open a Phase 2 trial of its experimental oral therapy Xanamem in boys with fragile X syndrome early next year. The ... Read more
October 23, 2020October 23, 2020
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its ... Read more
October 20, 2020October 20, 2020
Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest

Zygel, an experimental cannabidiol (CBD) gel, may lessen the extent of behavioral abnormalities in children with fragile X syndrome who have more severe disease, ... Read more
October 16, 2020October 16, 2020
UC Davis Establishing 4-Year Program for Students With Fragile X, Other Disabilities

The U.S. Department of Education awarded $2.1 million to the University of California, Davis, MIND Institute to create a four-year college program for students ... Read more
October 13, 2020October 13, 2020
Patrick Wild Centre Opens Website for Its Fragile X Registry

The Patrick Wild Centre at The University of Edinburgh launched an online portal for its fragile X syndrome registry on Oct. 10, European fragile X awareness ... 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

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

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

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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

New Streaming Channel Showcases Rare Disease Films

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

NIH Grant Supports Research Into Potential Targets for Fragile X Therapies

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

Blood Proteins May Help Measure Effectiveness of Metformin in Fragile X

Metformin Seen to Help Correct Defects in Neurons Lacking FMRP Protein

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

National Fragile X Awareness Month Is Underway

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

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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

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

Subscribe to our mailing list

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

New Streaming Channel Showcases Rare Disease Films

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

NIH Grant Supports Research Into Potential Targets for Fragile X Therapies

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

Blood Proteins May Help Measure Effectiveness of Metformin in Fragile X

Metformin Seen to Help Correct Defects in Neurons Lacking FMRP Protein

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

National Fragile X Awareness Month Is Underway

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

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

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