Updates

February 26, 2021February 26, 2021
UK Charity Sibs Offers Resources for Siblings of Fragile X Children

Sibs, a U.K. charity that supports siblings of all ages who have a brother or sister with disability, has presented several resources for siblings of children ... Read more
February 25, 2021February 25, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a ... Read more
February 23, 2021February 23, 2021
Parkinson’s Therapy May Hold Promise for Fragile X

Treatment with istradefylline — an approved therapy for Parkinson’s disease — normalized several molecular and functional nerve cell features of fragile X syndrome and improved ... Read more
February 22, 2021February 22, 2021
Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research ... Read more
February 19, 2021February 19, 2021
Nova Mentis Will Test Potential Psychedelic Treatment in Animal Model

Nova Mentis is expanding its preclinical pipeline to test a psychedelic treatment known as PLZ-1013 in an animal model of fragile X syndrome. The psychedelic ... 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. The ... Read more

Updates

UK Charity Sibs Offers Resources for Siblings of Fragile X Children

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Parkinson’s Therapy May Hold Promise for Fragile X

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Nova Mentis Will Test Potential Psychedelic Treatment in Animal Model

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Motor Problems Appear to Progress Faster in Men With Fragile X Premutation

Mitochondrial Dysfunction in Neuron-supporting Cells May Lead to Fragile X

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Fragile X Premutation Registry Opens to Eligible Adults Worldwide

Clues to Treating Fragile X Come From Altered Gene Regulation Process

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Women With Fragile X Premutations at Risk for Autistic Traits, Anxiety

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

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

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

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

UK Charity Sibs Offers Resources for Siblings of Fragile X Children

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Parkinson’s Therapy May Hold Promise for Fragile X

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Nova Mentis Will Test Potential Psychedelic Treatment in Animal Model

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Motor Problems Appear to Progress Faster in Men With Fragile X Premutation

Mitochondrial Dysfunction in Neuron-supporting Cells May Lead to Fragile X

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Fragile X Premutation Registry Opens to Eligible Adults Worldwide

Clues to Treating Fragile X Come From Altered Gene Regulation Process

Subscribe to our mailing list

Women With Fragile X Premutations at Risk for Autistic Traits, Anxiety

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

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

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