Updates

September 25, 2020September 25, 2020
Fragile X Girls Less Aware of Bullying, Study Suggests

School-aged girls with fragile X syndrome are less aware of bullying than children of the same age and intellectual abilities, a study suggests. These ... Read more
September 22, 2020September 22, 2020
Strong Placebo Effect Warrants Better Trial Design in Fragile X, Study Finds

Clinical improvements among participants receiving a placebo — known as the placebo effect — are common in clinical trials of fragile X syndrome, a ... Read more
September 18, 2020September 18, 2020
Hospitalization After Emergency Department Visit Common in Fragile X Patients, Study Finds

Almost half of emergency department visits for fragile X syndrome result in hospitalizations, according to a nationwide study in the U.S. The finding underscores ... Read more
September 15, 2020September 15, 2020
Zynerba, FDA to Discuss Next Steps for Zygel in Fragile X Treatment

The U.S. Food and Drug Administration (FDA) and Zynerba Pharmaceuticals will meet later this year to discuss the results of the CONNECT-FX trial and ... Read more
September 11, 2020September 11, 2020
Closing Leaky Mitochondria Halts Behavioral Problems in Fragile X, Study Suggests

Shortage of energy due to leaky mitochondria — the powerhouses of cells — contributes to behavioral abnormalities in fragile X syndrome, a study has ... 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

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

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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

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

Inflammation Not a Hallmark of Fragile X, Study Suggests

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

Coalition Will Address Racial Disparities in Rare Disease Communities

Genetic Screening Study Starts Recruiting Prospective Parents in Australia

Specific Piece of FMRP Protein Eases Hyperactivity in Mouse Model

Shionogi Acquires Tetra Therapeutics, Rights to Fragile X Treatment Candidate

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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

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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

Subscribe to our mailing list

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

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

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

Inflammation Not a Hallmark of Fragile X, Study Suggests

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

Coalition Will Address Racial Disparities in Rare Disease Communities

Genetic Screening Study Starts Recruiting Prospective Parents in Australia

Specific Piece of FMRP Protein Eases Hyperactivity in Mouse Model

Shionogi Acquires Tetra Therapeutics, Rights to Fragile X Treatment Candidate

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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