Updates

June 23, 2020June 23, 2020
Parent Language Intervention Eases Speech, Social Impairments in Fragile X, Trial Finds

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. ... Read more
June 22, 2020June 22, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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 ... Read more
June 19, 2020June 19, 2020
Inflammation Not a Hallmark of Fragile X, Study Suggests

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 ... Read more
June 12, 2020June 12, 2020
Trofinetide Safe and Effective at Higher Dose for Fragile X, Phase 2 Trial Finds

Oral treatment with trofinetide was well tolerated and, at a higher dose, eased social avoidance, anxiety, hyperactivity and other symptoms of fragile X syndrome ... Read more
June 10, 2020June 10, 2020
Coalition Will Address Racial Disparities in Rare Disease Communities

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 ... 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

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

Blocking GSK Alpha Enzyme Safely Reverses Fragile X Symptoms in Mouse Model

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Trial Results for Zygel, CBD Gel Treatment for Fragile X, Likely by Mid-year

OV101 Well-tolerated, Effective in Fragile X Patients, Phase 2 Trial Shows

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Expressive Language Sampling May Aid Research in Fragile X

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Clinical Symptoms and/or Family History of FXS Identify 96% of FXS Cases, Canadian Study Says

Urine microRNA Is Potential Biomarker for Fragile X, Early Study Suggests

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Israeli Clinic for Fragile X, One of World’s Largest, Adapting with COVID-19

NORD Partners with ‘Sing Me a Story’ for July 18-20 Living Rare Forum

Problems with Complex Speech, Talkativeness Seen as Common to Fragile X

Independence of Fragile X Patients Increases Until Their 30s, Then Starts to Decline, Study Says

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

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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

Blocking GSK Alpha Enzyme Safely Reverses Fragile X Symptoms in Mouse Model

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Subscribe to our mailing list

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Trial Results for Zygel, CBD Gel Treatment for Fragile X, Likely by Mid-year

OV101 Well-tolerated, Effective in Fragile X Patients, Phase 2 Trial Shows

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Lower Quality of Life Linked to Behavioral Problems in Fragile X Children, Parents Report

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Expressive Language Sampling May Aid Research in Fragile X

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Clinical Symptoms and/or Family History of FXS Identify 96% of FXS Cases, Canadian Study Says

Antipsychotic Medication Trifluoperazine Shows Promise for Fragile X, Study Finds

Urine microRNA Is Potential Biomarker for Fragile X, Early Study Suggests

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Israeli Clinic for Fragile X, One of World’s Largest, Adapting with COVID-19

NORD Partners with ‘Sing Me a Story’ for July 18-20 Living Rare Forum

Problems with Complex Speech, Talkativeness Seen as Common to Fragile X

Independence of Fragile X Patients Increases Until Their 30s, Then Starts to Decline, Study Says

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