News Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits

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

News Researchers Shed Light on Link Between IGF-1 and Potential Benefits in Rett Syndrome

Just Published, News Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases

News Newron Pharma Receives FDA Communication on STARS Trial; Results Expected Soon

News Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News MECP2 Mutation Type Predicts Rett Syndrome Bone Disease Severity, Study Finds

News FDA Grants Rare Pediatric Disease Designation to Trofinetide for Rett Syndrome

News Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News Restoring Wnt6 Protein Eases Behavioral, Motor Impairment, Mouse Study Suggests

News Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

News Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

News Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

News Newron Expands Rett Burden of Illness Survey to Europe and Australia

News Girls with Rett Need Better Speech Therapy and Communication Support in Schools, Survey Says

News FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News FDA Grants Fast Track Designation to Anavex 2-73 for Treatment of Rett Syndrome

News MECP2 Gene Mutations Alter Nerve Cells and Impair Learning, Mouse Study Finds

News Scientists Identify 2 New Genetic Mutations in 2 Iranian Girls With Rett Syndrome

News Study Suggests Critical Time Window to Treat Rett Syndrome

News Modification in Huntingtin Protein Prolongs Survival, Improves Lung Function in Rett Mouse Model, Study Finds

News DNA Sequencing Identifies Unusual Genetic Cause of Rett-like Disease in Young Girl

News Mutations in Only One MeCP2 Protein Form Leads to Milder Rett Symptoms in Boy, Report Indicates

News Anavex 2-73 Earns U.S. Patent for Neurodevelopmental Disorders, Including Rett Syndrome

News Low KCC2 Protein Levels May Underlie Developmental Defects in Rett Patients, Study Suggests

News New Genetic Causes of Rett Syndrome Found in Chinese Infants, Study Reports

News Stemonix and Atomwise Join Forces to Develop Small Molecules Targeting Rett Syndrome

News Parents of Rett Patients View Deep Brain Stimulation as Viable Option

News FDA, Newron Pharma to Meet in Step to Releasing Results of Sarizotan Pivotal Trial

News Gene Therapy Takes Center Stage at 2019 NORD Summit

News Altered Gut Microbiota Could Be Therapeutic Target in Rett Syndrome, Study Suggests

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

News Sarepta and StrideBio Partner to Advance Gene Therapies for Rett Syndrome, Other Disorders

News FDA Grants Rare Pediatric Disease Designation to Sarizotan for Treating Rett Syndrome

News Abnormal Breakdown of Certain Lipids May Underlie Rett Syndrome Development, Study Suggests

News FDA Grants Rare Pediatric Disease Designation to Anavex 2-73 for Rett Syndrome

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News New MECP2 Gene Mutations Found in Japanese Rett Syndrome Patients

News Animal Study Details Motor, Sensory and Respiratory Benefits of Anavex 2-73, Supporting Phase 2 Trials

News Newron Seeks Participants for Global Survey on Burden of Rett

News Phase 3 Trial of Trofinetide Recruiting Girls and Young Women with Rett Syndrome in US

News Treadmill Walking Exercises May Improve Gait Performance of Rett Syndrome Patients, Study Suggests

News Altered Protein Levels in Brain, Affecting Energy Metabolism, Underlies Rett Syndrome, Early Study Suggests

News Natural Antioxidant, CoQ10, as Dietary Supplement Eases Oxidative Stress in Rett Patients, Small Study Suggests

News New MECP2 Mutation May Lead to Atypical Rett Syndrome, Case Study Reports

News Rett Cells Have Defects in Energy Production, Greater Oxidative Stress, Study Suggests

News Social Media Campaigns, Purple Lighting Mark Rett Syndrome Awareness Month

News ‘Uptime’ Physical Activities Aid Life Quality and Walking Abilities in Rett Patients, Study Finds

News Resting Heart Rate Variability May Predict Reactivity to Tactile Stimuli, Study Suggests

News Reactivating Silenced X Chromosome Could Eventually Lead to Targeted Therapies, Researchers Say

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

News Rettsyndrome.org’s New CSO Has a Personal Connection to Disease

News Rare Case of Brothers with Rett Suggests Multiple Factors in Disease Course

News Preliminary Data From Anavex-2-73 Phase 2 Trial Suggest Safety, Efficacy

News Sensory Therapy Can Improve Hand Movements in Kids with Rett, Study Suggests

News EXCELLENCE Trial Will Test Anavex-2-73 in Children, Adolescents

News Existing Compounds Can Target KCC2 Protein to Ease Rett Symptoms, Early Study Reports

News X Chromosome Inactivation Not the Only Contributing Factor for Disease Severity in Female Rett Patients, Study Contends

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

News AveXis Delays Trial of Rett Gene Therapy, AVXS-201, to Conduct New Tests and Quality Control Checks

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

News Chemical Modifications That Accelerate KCC2 Activity May Ease Symptoms of Rett Syndrome, Mouse Study Indicates

News Monkeys With Rett Pay More Attention to Stronger Social Visual Stimuli, Study Finds

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

News Meta-Analysis of Genetic Studies Essential to Discover New Therapeutic Targets for Rett Syndrome, Reviewers Contend

News Algorithm to Measure Pupil Dilation, Heart Rate Could Help Detect Rett Earlier

News European Agency Recommends Orphan Designation for Anavex 2-73

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

News Thyroid Hormone Imbalance May Contribute to Rett Syndrome, Study Suggests

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

News Microswitch Intervention Program May Benefit Children with Rett Syndrome, Study Suggests

News Exosomes May Have Therapeutic Use in Rett Syndrome, Study Finds

News microRNAs Could Serve as Biomarkers of Rett’s Course, Study Suggests

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News Neurological Changes Present at Birth in Rett Syndrome, Mouse Study Finds

News Measures of Quality of Life Used for Children with Rett Also Valuable During Adulthood, Study Suggests

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

News First Patient Dosed in Phase 2 Extension Study of Anavex 2-73

News Subtle Signs of Rett Could Be Opportunity for Early Intervention, Study Says

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

News L-serine Improved Outcomes in Girl with Atypical Rett Syndrome, Case Report Says

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News Phase 2 AVATAR Study Testing Anavex 2–73 for Rett Syndrome Doses First Patient in Australia

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

News MeCP2 Optimized for Brain Delivery Holds Promise as Protein Replacement Therapy for RTT, Early Study Suggests

News Rettsyndrome.org Funds Research of Rett and MECP2 Syndromes

News Stem Cells of Rett Mice Prone to Arrested Growth, Less Able to Produce Energy, Study Shows

News Cannabidivarin Rescues Memory Deficits in Mouse Model of Rett, Study Shows

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

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

News Teeth Misalignment Linked to Swallowing Difficulties in Girls with Rett Syndrome, Study Says

News New Phase 2 Study on Anavex 2-73 for Rett Syndrome Begins in Australia

News #AANAM – Experimental Gene Therapy AVXS-201 Shows Promise to Treat Rett Syndrome in Preclinical Studies

News Overabundance of Nerve Fibers in the Skin May Affect Pain Sensitivity, Study Suggests

News Study in Rats Provides Clues to Nerve Cell Connectivity and Body Movement Control

News Mutations Causing Rett Syndrome Lead to Changes in Several Genes, Study Finds

News ‘Minds Wide Open’ Documentary Highlights Advances in Brain Research

News Early Protein Alterations May Drive the Development of Rett Biomarkers