News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News Challenging Behavior in Children with Fragile X Affects Maternal Mental Health and Mother-child Bond, Study Finds

News Fish Model of Fragile X Suggests Early Critical Time Window for Treatment, Study Shows

News Fragile X Impacts Children’s Cognitive Ability Most, Physical Functioning Least, Parents Report in Study

News Study Lends Support for Fragile X Genetic Screening in All Pregnant Women

News White Matter Lesions Among Brain MRI Pattern of FXTAS, Case Report Describes

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News Early Sound Exposure May Prevent Hypersensitivity to Noise in Fragile X, Mouse Study Suggests

News Fragile X Tremor-Ataxia Syndrome May Co-occur with Progressive Supranuclear Palsy, Case Study Reports

News Study Finds Higher-than-expected Prevalence of FMR1 Premutation Carriers Among Pregnant Women in Taiwan

News Healx Raises $56M to Launch Treatment Accelerator Program for Fragile X, Other Rare Diseases

News New Genetic Test May Help Identify Previously Undiagnosed Cases of Fragile X, Study Reports

News Grip Test Helps to Identify and Monitor Fragile X Tremor-Ataxia Syndrome in Adults, Study Suggests

News Study Links Autism Spectrum Disorders, Including Fragile X, to Low Cholesterol Levels

News Reduced Net-like Structures Around Nerve Cells May Cause Learning Deficits in FXS Patients, Mouse Study Suggests

News Autism Spectrum Disorder Worsens Motor Impairments of Fragile X Children, Study Reports

News Outpatient Genetic Counseling Improved Diagnostic Rates In Fragile X Patients, Others With Syndromic ASD, Study Finds

News Negative Emotions Early in Life Increase Risk for Anxiety in Boys with Fragile X, Study Suggests

News Derivatives of Alzheimer’s Disease Precursor May be Valuable Biomarkers of Disease Activity in Fragile X, Study Suggests

News Single Early-Life Seizure Leads to Fragile X-Like Behaviors Later in Life, Mouse Study Finds

News Diabetes Medicine Seen to Aid Language Skills, Ease Some Behaviors in Children in Review Study

News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News Caregivers Validate Scale Used to Assess Behaviors of Children with Fragile X

News Anxiety, Common to Fragile X and Like Disorders, Might Be Eased with CBD Gel, Study Finds

News Signaling Cascade Discovered in Fruit Flies May Be Promising Target for FXS Therapy

News AGG Triplets in FMR1 Linked to Number of CGG Repeats, Consanguinity, Study Finds

News Taurine Supplementation Improves Testes Function in Fragile X Mice, Early Study Suggests

News Antipsychotics Most Common Prescription for Aggressive Behaviors in Fragile X Patients, Study Reveals

News Profile of Premutation Carriers Reflects Increased Risk of Mental Health Disorders, Study Says

News ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News Revised Scales Better – But Not Enough – for Diagnosing Autism in Fragile X, Study Finds

News Children with FXS Have Difficulty Fixing Visual Attention, Study Finds

News Adequate Preventive Care for Young Patients Linked to Health, Demographics, Study Finds

News Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News Studies to Test Diabetes Treatment, Metformin, on Fragile X Patients Now Enrolling in US and Canada

News Zynerba’s Cannabidiol Gel Eases Anxiety and Other Behaviors in Youth with Fragile X, Trial Shows

News Deficits in Language Processing Seen in FMR1 Premutation Carriers

News Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News FMRP Protein Key in Nerve Cell Production From Stem Cells After Birth, Study Suggests

News Posture Impairment May be Early Sign of FXTAS in Premutation Carriers

News Unusual Behaviors of Fragile X Stop with OV101 Treatment, Study in Mouse Model Reports

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News Social Avoidance in Infancy Predicts Autism Severity in Boys with Fragile X Syndrome, Study Says

News Report: Hummingbird Sign Links FXTAS, Progressive Supranuclear Palsy

News Alzheimer’s Therapy, Donepezil, Seen to Lessen Arousal of Cognitive Brain Area in Fragile X Patients in Study

News World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

News Hyper-Excited Neurons Disrupt Perception in Fragile X Syndrome, Fruit Fly Study Suggests

News Restoring FMR1 Activity and Protein Production in Cells May Be Possible, Early Study Says

News NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News Long Noncoding RNAs May Be New Target for Diagnosis, Treatment of Fragile X Spectrum Disorders

News Social Avoidance Behaviors Emerge in Infancy, Increase During Childhood, Study Finds

News ‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

News FXTAS Should be Considered in the Diagnosis of Patients With Essential Tremor Who Show Certain Signs, Study Says

News Early Short-term Lovastatin Treatment May Correct Learning Deficits in Fragile X Syndrome, Rat Study Suggests

News Self-Injury and Aggressive Behaviors Highly Persistent Among Males, Longitudinal Study Says

News Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

News Diabetes Medicine Metformin May Improve Cognition, Behavior Symptoms in Fragile X Syndrome Patients, Case Study Reports

News Modulation of Potassium Channels May Treat Symptoms in FXS, Study Finds

News Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

News Cannabidiol Gel ZYN002 Granted Fast Track Status for Treatment of Behavioral Symptoms in Fragile X Syndrome

News Confluence Gets $200K from University-affiliated Venture Fund to Develop Fragile X Treatment

News Cognitive Training Program Provides Benefits for Young Fragile X Syndrome Patients, Trial Finds

News CRISPR-Gold, Using Non-viral Carrier, Edits Fragile X Gene in Mice to Ease Exaggerated Behaviors

News Mindfulness Program Run on App May Help Ease Stress in Women with Fragile X Children, Study Says

News Telehealth Is Feasible, Effective Way to Treat Behavioral Issues in FRX, Study Shows

News Patient’s Age, Family Income are Factors for Accessing Healthcare, FXS Caregivers Survey Reveals

News Green Tea Compound Significantly Improves Cognitive Function in Fragile X Patients, Study Finds

News Early Social Avoidance Can Predict Autism but Not Anxiety in Boys with FXS, Study Says

News Pediatricians Unfamiliar With Guidelines for Genetic Testing of Children With Global Developmental Delay, Study Finds

News Mavoglurant Rescues Specific Neural Networks in FXS Mice, Study Finds

News Qualitative Study Identifies Major Concerns of Young Adults With Fragile X and Their Parents

News Combo Treatment Can Reactivate FMR1 Gene in Fragile X, Study Shows

News Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

News Decisional Capacity of Women With Fragile X Surpasses That of Men, Study Says

News Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News Current FXTAS Diagnostic Criteria Show Low Accuracy, Study Finds

News NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

News Zynerba Pharmaceuticals Receives U.S. Patent for Cannabidiol Gel ZYN002

News Understanding of How Females Inherit Traits May Shed Light on Fragile X, Rett Syndromes, Study Says

News WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

News Vienna to Host RARE2019 Meeting on Rare Diseases

News Information, Support May Persuade Parents of FXS Children to Join Trials

News Defective Mitochondria Could Explain Neurological Impairment in Fragile X, Research Shows

News Mainstreaming Benefits Girls with Fragile X Outside Classroom, Study Suggests

News Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

News Language Use Differs Between Boys with Both Autism and FXS and Autism Alone, Study Shows

News FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says

News Anxiety, Learning and Behavior Issues Top Concerns in Fragile X Syndrome, Survey Shows

News Novartis’ Mavoglurant May Improve Visual Attention, Pupil Reactivity in Fragile X, Study Says

News Inhibiting HDAC6 Enzyme Has Potential for Treating FRX, Mouse Study Finds

News Effort Afoot to Expand ‘Early Check’ Newborn Screening for Fragile X in the U.S.

News Tetra Discovery and Shionogi Team Up to Further Study BPN14770 for Potential in Fragile X, Other Disorders

News FMR1 Mutations Linked to Early Atypical Temperament, Study Shows

News Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News Cannabidiol Gel ZYN002 Shows Sustained Benefits in Children and Teens with Fragile X, Zynerba Reports

News Ability to Form Words, Sentences Linked with Autism Spectrum Disorder Severity in Young Males with Fragile X

News Mavoglurant Safe, Well-tolerated Long-term in Adolescents and Adults with Fragile X Syndrome, Studies Show