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

News Social-Environmental Factors Contribute to Self-Injury Behavior in Fragile X, Study Finds

News Study Examines Behavioral Traits of Fragile X Patients Without Autism

News Children With Fragile X Show Impaired Use of Gestures, Study Suggests

News New Genetic Test Better at Predicting If a Child Will Be Born with Fragile X

News Mouse Model Provides More Insight into Impaired Spacial Processing in Fragile X Syndrome

News FRAXA Funds 2 Projects to Tackle Potential New Fragile X Therapies

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News Cognitive Behavioral Therapy May Help Kids with Autism, Including Fragile X, Manage Emotions

News Researchers Awarded $100K Grant to Study Reversal of Fragile X Symptoms

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News Hypertension Medicine May Have Application in Fragile X Syndrome, Mouse Study Shows

News CDC Report Shows Highest Autism Prevalence Rates Since 2000 in US

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News Fostering Academic, Living Skills in Early Childhood Increases Fragile X Patients’ Adult Independence

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News Experts Update Treatment Recommendations for Fragile X-Associated Tremor/Ataxia Syndrome

News Researcher Earns $300K Hartwell Award to Develop Potential Fragile X Syndrome Therapy

News Fragile X Syndrome Linked to Impaired Brain White Matter Development, Researchers Say

News Lysogene and French Scientist Partner to Develop Gene Therapy for Fragile X Syndrome

News #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News Brain Activity Alterations Cause Hearing Problems in Fragile X Syndrome, Mouse Study Finds

News Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases

News Researcher Develops Fitness Programs to Help People with Intellectual Disabilities

News U-Lace, FRAXA Partner to Help Fund Research for Fragile X Syndrome

News Children with Autism, Intellectual Disability Likely to Be Given Antipsychotics, Study Finds

News FDA Grants Orphan Drug Status to Investigational Therapy BPN14770 for Fragile X Syndrome

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Increased Protein Synthesis Is a Biomarker of Fragile X Syndrome, Study Suggests

News FRAXA, Healx Collaboration Identifies Therapies that Could Be Repurposed for Fragile X

News FDA Grants Fast Track Designation to OV101 for Fragile X

News FDA Decision Clears Way for Phase 2 Clinical Trial of BPN14770

News Zynerba to Start Trial of Its Fragile X Syndrome Therapy in Mid-2018

News Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

News ‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event

News Fragile X Syndrome Neurons’ Function Restored with CRISPR/Cas9 Genome Editing

News DNA Mutation Linked to Impaired Motor Skills in Children on the Autism Spectrum

News Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

News US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs

News Genomics Collaborative Aims to Find New Precision Medicine Methods

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

News Mutation in TSC2 Gene Linked to Low FMRP Protein Levels in Early Study

News Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

News GW Announces Increased Focus on Developing GWP42006 Therapy for Fragile X, Other Disorders

News Slower Manual Movement Seen in Fragile X Premutation Carriers Before Disease Onset

News BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

News UC Davis Mind Institute Recruiting Fragile X Patients for Spoken Language Studies

News Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

News Loss of FMR1 Gene Linked to Lower Reproductive Capacity in Mice

News Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

News Rapamycin Fails to Reverse Fragile X-related Behavior, Study in Mice Shows

News Study Links Missing Protein to Fragile X Patients’ Hypersensitivity to Sound

News Inflexible Learning Disability in Fragile X Syndrome Explained in New Mouse Study

News NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

News, Social Clips Lack of Nerve Cell Coordination May Cause Fragile X Cognitive Inflexibility