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

News World’s Largest Newborn Screening Study to Include Fragile X Syndrome

News Exposure to Pesticides Might Increase Severity of Fragile X Spectrum Disorders, Study Finds

News, Social Clips Cognitive Function Affects Gait, Motor Function in FXTAS, Study Finds

News Fragile X Added to Newborn Screening in North Carolina Through New Study

News #NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

News Changes in Calcium Dynamics Within Nerve Cells May Be Biomarker for Fragile X, Study Reports

News Social Anxiety Traits Overlap Between Fragile X Syndrome, Autism Spectrum Disorder

News Therapeutics Targeting cAMP Signaling Can Help Improve Sensory Perception in Patients with Fragile X Syndrome

News Genome Folded Incorrectly in Fragile X Syndrome, Other Neurological Conditions, Study Finds

News Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News Irregular Visual Perception in a Mouse Model of Fragile X Syndrome Could be Treated using Designer Therapies

News Simvastatin Does Not Show Same Brain Benefits for Fragile X as Lovastatin, Study Reports

News Allopregnanolone May Help Improve Cognitive Function in Fragile X-Associated Tremor/Ataxia Syndrome, Study Suggests

News Cellular ‘Cleaning System’ a Possible Therapeutic Target for Fragile X, Mouse Study Finds

News Researchers Identify Potential New Fragile X Therapeutic Target Called Kv1.2

News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News Human iPSCs Beneficial for Study of Autism Spectrum Disorders, but Limitations Still Exist, Review Says

News IL-6 Molecule May Be Behind Abnormal Nerve Cell Communication in Fragile X, Study Suggests

News New Roles for FMRP and p53 Proteins in Fragile X Identified in Mouse Study

News Neurotrope, Nemours to Begin Bryostatin-1 Clinical Trial in Fragile X Children

News Pilot Study Finds Behavioral Training Can Improve ‘Social Gaze’ Skills in Boys with Fragile X

News Production of Autism-related Large Proteins Abnormal in Fragile X, Fruit Fly Study Shows

News Increased Fragile X Awareness Can Allay Delayed Diagnosis, Small Study Suggests

News Mouse Study of Fragile X Reflects Patients’ Abnormal Sleep Structure

News Tool Reactivates Gene in Stem Cells Harboring Fragile X Mutation, Study Shows

News Small Molecule That Regulates Nerve Cell Communication Shows Potential for Fragile X Treatment, Study Says

News New Therapeutic Strategy Minimizes Fragile X Cognitive, Behavioral Defects in Mice

News Phase 2 Trial Data Show Therapeutic Benefits of Investigational Cannabidiol Gel ZYN002 in Children with Fragile X Syndrome

News Ovid Starts Phase 2 ROCKET Trial Testing OV101 in Adolescents with Fragile X Syndrome

News Zynerba Starts Pivotal Trial of ZYN002, Investigational Cannabidiol Gel for Fragile X Treatment

News Aripiprazole Improved Irritable Behavior in Patients With Fragile X Syndrome, Phase 2 Study Shows

News Invitae Launches Comprehensive Carrier Screening for Genetic Disorders, Including Fragile X

News Modification of DNA-binding Protein May Be New Therapeutic Target for Fragile X, Mouse Study Suggests

News Gene Editing Tool CRISPR-Gold May Reduce Autism-Related Behaviors, Fragile X Mouse Model Shows

News Possible Skin Biomarker to Diagnose Fragile X-linked Syndrome Identified in Study